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What changed in uniQure N.V.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of uniQure N.V.'s 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+528 added−499 removedSource: 10-K (2026-03-02) vs 10-K (2025-02-27)

Top changes in uniQure N.V.'s 2025 10-K

528 paragraphs added · 499 removed · 391 edited across 2 sections

Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

250 edited+83 added−38 removed304 unchanged
Biggest changeFDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in NfL measured in CSF may serve as supportive evidence of therapeutic benefit in the application for accelerated approval. ​ There are numerous factors that could impede or otherwise negatively impact our further development of AMT-130, including, but not limited to, potential patient safety issues; our failure to demonstrate sufficient clinical efficacy or durability of response data to warrant further development or accelerated approval by FDA, EMA or any other regulatory authority; our ability to achieve alignment with FDA and other regulatory authorities on the primary statistical analysis plan and CMC requirements to support registration and the timing of such regulatory alignment; the results from future interim data readouts from our Phase I/II trial, including the three-year follow-up data from treated patients and safety and tolerability data from the third cohort; any requirement for a Phase III confirmatory study; the timing and resources associated with our planned marketing applications; our ability to successfully commercialize AMT-130 should we choose to do so without a partner; challenges with potential development or commercial partners, should we choose to pursue further development or commercialization of AMT-130 with a partner; and our ability to fund the further development and commercialization of the AMT-130 program. ​ Any one or combination of these factors could force us to halt or discontinue the ongoing clinical trials of AMT-130 or related commercialization efforts.
Biggest changeSee also “ —Risks Related to Regulatory Approval of Our Products .” ​ ​ 40 ​ Table of Contents There are numerous factors that could impede or otherwise negatively impact our further development of AMT-130, including, but not limited to: potential patient safety issues; our failure to demonstrate sufficient clinical efficacy or durability of response data to warrant further development or accelerated or regular approval by the FDA, European Commission, MHRA or any other regulatory authority; shifting FDA standards for an adequate and well-controlled trial in a rare disease population under clinical investigation; any inability to achieve alignment regarding an accelerated approval pathway for AMT-130 with the FDA, and the timing of such regulatory alignment and other factors impacting our interactions with the FDA, which may be outside of our control; our ability to align on an approval pathway for AMT-130 with other regulatory agencies, such as the European Commission or MHRA; our current beliefs regarding the further development of and approval pathway for AMT-130, which are based on our interpretation of communications and interactions with the FDA to date, and the success of our efforts to address such communications and interactions; the results from future interim or topline data readouts from our Phase I/II trials, including as additional patient data becomes available; any requirement for additional studies to obtain approval and for a Phase III confirmatory study; the timing and resources associated with our planned marketing applications; our ability to successfully commercialize AMT-130 should we choose to do so without a partner; challenges with potential development or commercial partners, should we choose to pursue further development or commercialization of AMT-130 with a partner; and our ability to fund the further development and commercialization of the AMT-130 program. ​ Any one or combination of these factors could force us to halt or discontinue the ongoing clinical trials of AMT-130 or related commercialization efforts or could prevent us from obtaining marketing approval within the predicted timeframes or at all.
Accordingly, the results from our open-label trials, including early indications of potential efficacy, may not be predictive of future clinical trial results. Early evidence of slowing of disease progression in our AMT-130 clinical trial may not be predictive of continued evidence of potential efficacy as we continue to collect follow-up data from patients enrolled in the trial.
Accordingly, the results from our open-label trials, including early indications of potential efficacy, may not be predictive of future clinical trial results. Early evidence of slowing disease progression in our AMT-130 clinical trial may not be predictive of continued evidence of potential efficacy as we continue to collect follow-up data from patients enrolled in the trial.
In addition to AMT-130, we are also developing other investigational gene therapies, including AMT-260 for the treatment of mTLE, AMT-162 for the treatment of SOD1-ALS and AMT-191 for the treatment of Fabry disease.
In addition to AMT-130, we are also developing other investigational gene therapies, including AMT-260 for the treatment of MTLE, AMT-191 for the treatment of Fabry disease, and AMT-162 for the treatment of SOD1-ALS.
Depending on the products, whether two products are ultimately considered to be the same may be determined by FDA on a case-by-case basis, making it difficult to make predictions regarding when the FDA might be able to make an approval of a product effective and whether periods of exclusivity will effectively block competitors seeking to market products that are the same or similar to ours for the same intended use.
Depending on the products, whether two products are ultimately considered to be the same may be determined by the FDA on a case-by-case basis, making it difficult to make predictions regarding when the FDA might be able to make an approval of a product effective and whether periods of exclusivity will effectively block competitors seeking to market products that are the same or similar to ours for the same intended use.
Moreover, if a company obtains FDA approval for a product via the accelerated approval pathway, the company would be required to conduct a post-marketing confirmatory trial to verify and describe the clinical benefit in support of full approval. FDA can, and frequently does, require that this confirmatory trial be commenced prior to FDA granting a product accelerated approval.
Moreover, if a company obtains FDA approval for a product via the accelerated approval pathway, the company would be required to conduct a post-marketing confirmatory trial to verify and describe the clinical benefit in support of full approval. The FDA can, and frequently does, require that this confirmatory trial be commenced prior to the FDA granting a product accelerated approval.
Our ability to generate revenues from our product candidates will depend on the successful development and eventual commercialization of our product candidates, whether we choose to pursue further development or commercialization our product candidates alone or with a partner.
Our ability to generate revenues from our product candidates will depend on the successful development and eventual commercialization of our product candidates, whether we choose to pursue further development or commercialization of our product candidates alone or with a partner.
In particular, have in the past been subject to allegations of Sarbanes-Oxley whistleblower retaliation and employment discrimination and retaliation, and we may in the future be subject to additional claims of non-compliance with similar or other laws and regulations.
In particular, we have in the past been subject to allegations of Sarbanes-Oxley whistleblower retaliation and employment discrimination and retaliation, and we may in the future be subject to additional claims of non-compliance with similar or other laws and regulations.
Such actions could also adversely restrict our business and operations. There could also be changes in FDA’s approval standards that could impact our ability to obtain BLA approval and market our product candidates within the currently anticipated timeframes or otherwise impact the competitive market for our product candidates.
Such actions could also adversely restrict our business and operations. There could also be changes in the FDA’s approval standards that could impact our ability to obtain BLA approval and market our product candidates within the currently anticipated timeframes or otherwise impact the competitive market for our product candidates.
Such changes may necessitate the conduct of additional development work, including preclinical and clinical trials, and manufacturing development. By example, for products intended for rare and serious diseases with unmet medical needs, FDA is authorized to exercise regulatory flexibility when making a medical risk-benefit judgment.
Such changes may necessitate the conduct of additional development work, including preclinical and clinical trials, and manufacturing development. By example, for products intended for rare and serious diseases with unmet medical needs, the FDA is authorized to exercise regulatory flexibility when making a medical risk-benefit judgment.
Moreover, there could be changes in the federal workforce and agency policies that may result in regulatory delays, including with respect to FDA’s review of marketing applications and other submissions, and that may impact the ability to communicate with and obtain guidance from the agencies.
Moreover, there could be changes in the federal workforce and agency policies that may result in regulatory delays, including with respect to the FDA’s review of marketing applications and other submissions, and that may impact the ability to communicate with and obtain guidance from the agencies.
In addition, payment of future cash dividends may be made only if our shareholders’ (deficit) / equity exceeds the sum of our paid-in and called-up share capital plus the reserves required to be maintained by Dutch law or by our articles of association.
In addition, payment of future cash dividends may be made only if our shareholders’ equity / (deficit) exceeds the sum of our paid-in and called-up share capital plus the reserves required to be maintained by Dutch law or by our articles of association.
In connection with the closing of the Lexington Transaction in July 2024, we and Genezen entered into certain additional agreements, including a commercial supply agreement pursuant to which Genezen will manufacture and supply our requirements of HEMGENIX® pursuant to our manufacturing and supply obligations to CSL Behring, and development and other manufacturing services agreement pursuant to which Genezen will manufacture, supply and provide certain development services to support the requirements of our investigational gene therapy programs and for other discretionary services related to the manufacture of HEMGEMIX®, along with other customary agreements.
In connection with the July 2024 closing of the Lexington Transaction, we and Genezen entered into certain additional agreements, including a commercial supply agreement pursuant to which Genezen will manufacture and supply our requirements of HEMGENIX pursuant to our manufacturing and supply obligations to CSL Behring, and development and other manufacturing services agreement pursuant to which Genezen will manufacture, supply and provide certain development services to support the requirements of our investigational gene therapy programs and for other discretionary services related to the manufacture of HEMGENIX, along with other customary agreements.
Orphan drug exclusivity may be lost for a number of reasons, including, but not limited to if the FDA or the EMA determines that the request for designation was materially defective, or if the manufacturer is unable to assure sufficient quantity of the drug to meet the needs of patients with the rare disease or condition.
Orphan drug exclusivity may be lost for a number of reasons, including, but not limited to if the FDA, the EMA or the MHRA determines that the request for designation was materially defective, or if the manufacturer is unable to assure sufficient quantity of the drug to meet the needs of patients with the rare disease or condition.
We expect that additional U.S. federal healthcare reform measures will be adopted in the future, any of which could limit coverage and/or the amounts that the U.S. federal government will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures and could seriously harm our business. 53 ​ Table of Contents Individual states in the U.S. have also increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
We expect that additional U.S. federal healthcare reform measures will be adopted in the future, any of which could limit coverage and/or the amounts that the U.S. federal government will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures and could seriously harm our business. ​ ​ 56 ​ Table of Contents Individual states in the U.S. have also increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
We also anticipate that many or all our gene therapy product candidates may provide long-term, and potentially curative benefit, with a single administration. This is a different paradigm than that of many other pharmaceutical therapies, which often require an extended course of treatment or frequent administration.
We also anticipate that many of our gene therapy product candidates may provide long-term, and potentially curative benefit, with a single administration. This is a different paradigm than that of many other pharmaceutical therapies, which often require an extended course of treatment or frequent administration.
We are highly dependent on hiring, training, retaining, and motivating key personnel to lead our research and development, clinical operations, and manufacturing efforts. Although we have entered into employment agreements with our key personnel, each of them may terminate their employment subject to the notice provisions in such agreements.
We are highly dependent on hiring, training, retaining, and motivating key personnel to lead our research and development, clinical operations, commercialization and manufacturing efforts. Although we have entered into employment agreements with our key personnel, each of them may terminate their employment subject to the notice provisions in such agreements.
If the preliminary or interim data that we report differ from final results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, product candidates may be harmed, which could seriously harm our business.
If the preliminary, interim or topline data that we report differ from final results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, product candidates may be harmed, which could seriously harm our business.
The FDA and the EMA have issued various guidance documents pertaining to gene therapy products, which are and will be applicable to our product candidates. The close regulatory scrutiny of gene therapy products may result in delays and increased costs and may ultimately lead to the failure to obtain approval for any gene therapy product.
The FDA, the EMA and MHRA have issued various guidance documents pertaining to gene therapy products, which are and will be applicable to our product candidates. The close regulatory scrutiny of gene therapy products may result in delays and increased costs and may ultimately lead to the failure to obtain approval for any gene therapy product.
The relatively small market size for orphan indications and the potential for long-term therapeutic benefit from a single administration present challenges for pricing review and negotiation of our product candidates for which we may obtain marketing authorization. Most of our product candidates target rare diseases with relatively small patient populations.
The relatively small market size for indications and the potential for long-term therapeutic benefit from a single administration present challenges for pricing review and negotiation of our product candidates for which we may obtain marketing authorization. Most of our product candidates target rare diseases with relatively small patient populations.
The GDPR, together with the national legislation of the UK (including the Data Protection Act 2018) and EU member states governing the processing of personal data, impose strict obligations and restrictions on the ability to collect, use, analyze and transfer personal information, including health data from clinical trials and adverse event reporting.
The GDPR, together with the national legislation of Switzerland, the UK (including the Data Protection Act 2018) and EU member states governing the processing of personal data, impose strict obligations and restrictions on the ability to collect, use, analyze and transfer personal information, including health data from clinical trials and adverse event reporting.
An RMAT designation is designed to accelerate approval for regenerative advanced therapies. Priority review designation is intended to accelerate the FDA marketing application review timeframe for drug products that treat a serious condition and that, if approved, would provide a significant improvement in safety or effectiveness.
An RMAT designation is designed to accelerate approval timelines for regenerative advanced therapies. Priority review designation is intended to accelerate the FDA marketing application review timeframe for drug products that treat a serious condition and that, if approved, would provide a significant improvement in safety or effectiveness.
It is possible that whether and how FDA exercises any such regulatory flexibility, including with respect to specialized pathways, such as accelerated approval, may change, which could impact our ability to obtain approval for AMT-130 or any of our product candidates.
It is possible that whether and how the FDA exercises any such regulatory flexibility, including with respect to specialized pathways, such as accelerated approval, may change, which could impact our ability to obtain approval for AMT-130 or any of our product candidates.
Interim or preliminary data also remain subject to regulatory audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. As a result, preliminary or interim data should be viewed with caution until the final data are available.
Interim and preliminary data also remain subject to regulatory audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. As a result, preliminary, interim or topline data should be viewed with caution until the final data are available.
National governments and health service providers have different priorities and approaches to the delivery of health care and the pricing and reimbursement of products in that context. In general, however, the healthcare budgetary constraints in most EU member states have resulted in restrictions on the pricing and reimbursement of medicines by relevant health service providers.
National governments and health service providers have different priorities and approaches to the delivery of health care and the pricing and reimbursement of products in that context. In general, however, the healthcare budgetary constraints in most EU member states and in the UK have resulted in restrictions on the pricing and reimbursement of medicines by relevant health service providers.
In addition, legal proceedings relating to intellectual property claims, with or without merit, are unpredictable and generally expensive and time-consuming and is likely to divert significant resources from our core business, including distracting our technical and management personnel from their normal responsibilities.
In addition, legal proceedings relating to intellectual property claims, with or without merit, are unpredictable and generally expensive and time-consuming and are likely to divert significant resources from our core business, including distracting our technical and management personnel from their normal responsibilities.
These reforms could reduce the ultimate demand for our product candidates or put pressure on our product pricing and could seriously harm our business. In the EU, similar political, economic, and regulatory developments may affect our ability to profitably commercialize our product candidates, if approved.
These reforms could reduce the ultimate demand for our product candidates or put pressure on our product pricing and could seriously harm our business. In the EU and UK, similar political, economic, and regulatory developments may affect our ability to profitably commercialize our product candidates, if approved.
The facilities used by Genezen and our other contract manufacturers are subject to FDA inspections, including after we submit a BLA. We are completely dependent on Genezen and our other contract manufacturers to execute on our manufacturing processes for HEMGENIX® and other product candidates and for compliance with cGMP requirements.
The facilities used by Genezen and our other contract manufacturers are subject to FDA inspections and by other authorities, including after we submit a BLA. We are completely dependent on Genezen and our other contract manufacturers to execute on our manufacturing processes for HEMGENIX and other product candidates and for compliance with cGMP requirements.
These oppositions and future patent oppositions may result in loss of scope of some claims or the entire patent and, with respect to our rights under the CSL Agreement, could affect CSL Behring’s successful commercialization of HEMGENIX® and, in turn, could negatively impact our financial position.
These oppositions and future patent oppositions may result in loss of scope of some claims or the entire patent and, with respect to our rights under the CSL Behring Agreement, could affect CSL Behring’s successful commercialization of HEMGENIX and, in turn, could negatively impact our financial position.
Interim or preliminary results from our clinical trials may change as more data become available, as such data are subject to regulatory audit and verification procedures, and regulatory review, which could result in material changes in the final results and conclusions.
Interim, topline or preliminary results from our clinical trials may change as more data become available, as such data are subject to regulatory audit and verification procedures, and/or regulatory review, which could result in material changes in the final results and conclusions.
We currently rely and expect to continue to rely on third parties to manufacture our product candidates, and these third parties may not perform satisfactorily or may fail to comply with these regulations or maintain these approvals. The manufacturing of our products and product candidates is subject to significant government regulation.
The manufacturing of our products and product candidates is subject to significant government regulations and approvals. We currently rely and expect to continue to rely on third parties to manufacture our product candidates, and these third parties may not perform satisfactorily or may fail to comply with these regulations or maintain these approvals.
We have based our current estimates of our financing requirements on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. Adequate capital may not be available to us when needed or may not be available on acceptable terms.
We have based our current estimates of our financing requirements on assumptions that may prove to be wrong, and we could use our capital resources sooner than we expect. ​ Adequate capital may not be available to us if and when needed or may not be available on acceptable terms.
We do not maintain key person insurance for any of our senior management or employees. The loss of the services of our key employees could impede the achievement of our research and development objectives and seriously harm our ability to successfully implement our business strategy.
We do not maintain key person insurance for any of our senior management or employees. The loss of the services of our key employees could impede the achievement of our research and development and commercialization objectives and seriously harm our ability to successfully implement our business strategy.
Experiences with existing gene therapies, including any emergent adverse effects, could also impact how the FDA and the EMA view our products and product candidates, making it harder to obtain or maintain regulatory approvals.
Experiences with existing gene therapies, including any emergent adverse effects, could also impact how the FDA, the EMA and MHRA view our products and product candidates, making it harder to obtain or maintain regulatory approvals.
All the risks relating to product development, regulatory approval and commercialization described herein also apply to the activities of any development collaborators. 64 ​ Table of Contents Risks Related to Our Intellectual Property We rely on licenses of intellectual property from third parties, and such licenses may not provide adequate rights, may be open to multiple interpretations or may not be available in the future on commercially reasonable terms or at all, and our licensors may be unable to obtain and maintain patent protection for the technology or products that we license from them.
All the risks relating to product development, regulatory approval and commercialization described herein also apply to the activities of any development collaborators. 68 ​ Table of Contents Risks Related to Our Intellectual Property We rely on licenses of intellectual property from third parties, and such licenses may not provide adequate rights, may be open to multiple interpretations or may not be available in the future on commercially reasonable terms or at all, and our licensors may be unable to obtain and maintain patent protection for the technology or products that we license from them.
We also make assumptions, estimations, calculations, and conclusions as part of our preliminary or interim analyses of data, and we may not have received or had the opportunity to evaluate all data at that time.
We also make assumptions, estimations, calculations, and conclusions as part of our preliminary, interim or topline analyses of data, and we may not have received or had the opportunity to evaluate all data at that time.
An adverse result in any litigation proceeding could put one or more of our patents at risk of being invalidated, maintained in a more narrowly amended form or interpreted narrowly. 66 ​ Table of Contents Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, increase our operating losses, reduce available resources, and could distract our technical and management personnel from their normal responsibilities.
An adverse result in any litigation proceeding could put one or more of our patents at risk of being invalidated, maintained in a more narrowly amended form or interpreted narrowly. ​ ​ 70 ​ Table of Contents Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, increase our operating losses, reduce available resources, and could distract our technical and management personnel from their normal responsibilities.
By example, FDA issued a number of guidance documents, and continues to issue guidance documents, on human gene therapy development, one of which was specific to human gene therapy for hemophilia, one that was specific to neurodegenerative diseases, and another of which was specific to rare diseases.
By example, the FDA issued a number of guidance documents, and continues to issue guidance documents, on human gene therapy development, one of which was specific to human gene therapy for hemophilia, one that was specific to neurodegenerative diseases, and another of which was specific to rare diseases.
If one or more of these analysts ceases coverage of our company or fails to publish reports on us regularly, demand for our shares could decrease, which might cause our share price and trading volume to decline. 81 ​ Table of Contents If we do not achieve our projected development and financial goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and, as a result, our share price may decline.
If one or more of these analysts ceases coverage of our company or fails to publish reports on us regularly, demand for our shares could decrease, which might cause our share price and trading volume to decline. ​ ​ ​ ​ 87 ​ Table of Contents If we do not achieve our projected development and financial goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and, as a result, our share price may decline.
Termination of these agreements or reduction or elimination of our rights under these agreements may result in our having to negotiate new or amended agreements with less favorable terms or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology. 65 ​ Table of Contents If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired.
Termination of these agreements or reduction or elimination of our rights under these agreements may result in our having to negotiate new or amended agreements with less favorable terms or cause us to lose our rights under these agreements, including our rights to important intellectual property or technology. ​ ​ ​ ​ ​ 69 ​ Table of Contents If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired.
These laws may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations. 74 ​ Table of Contents We anticipate that the PPACA, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and additional downward pressure on pricing and the reimbursement our customers may receive for our products, and increased manufacturer rebates.
These laws may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations. ​ ​ ​ ​ 78 ​ Table of Contents We anticipate that the PPACA, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and additional downward pressure on pricing and the reimbursement our customers may receive for our products, and increased manufacturer rebates.
Failure to confirm favorable results from earlier trials by demonstrating the safety and effectiveness of our products in later-stage clinical trials with larger patient populations could have a material adverse effect on our business, financial condition, and results of operations. 43 ​ Table of Contents Additionally, we are currently conducting and may in the future conduct clinical trials that utilize an “open-label” trial design.
Failure to confirm favorable results from earlier trials by demonstrating the safety and effectiveness of our products in later-stage clinical trials with larger patient populations could have a material adverse effect on our business, financial condition, and results of operations. 45 ​ Table of Contents Additionally, we are currently conducting and may in the future conduct clinical trials that utilize an “open-label” trial design.
The delivery of healthcare in the EU, including the establishment and operation of health services and the pricing and reimbursement of medicines, is almost exclusively a matter for national, rather than EU, law and policy.
The delivery of healthcare in the EU and UK, including the establishment and operation of health services and the pricing and reimbursement of medicines, is almost exclusively a matter for national, rather than EU, law and policy.
The UK has, following its exit from the EU, substantially adopted the EU General Data Protection Regulation into its domestic law through the UK General Data Protection Regulation (collectively with the EU General Data Protection Regulation, and related EU and UK e-Privacy laws, the “GDPR”).
The UK has, following its exit from the EU, substantially adopted the EU General Data Protection Regulation into its domestic law through the UK General Data Protection Regulation (collectively with the EU General Data Protection Regulation, and related EU, Swiss, and UK data protection and e-Privacy laws, the “GDPR”).
If we are unable to continue to attract and retain high quality personnel, our ability to pursue our business may be harmed and our growth strategy may be limited. 79 ​ Table of Contents We may be adversely affected by unstable market and economic conditions and potential macroeconomic effects, such as inflation, new or increased tariffs and higher interest rates, which may negatively impact our business, financial condition and share price.
If we are unable to continue to attract and retain high quality personnel, our ability to pursue our business may be harmed and our growth strategy may be limited. ​ ​ 85 ​ Table of Contents We may be adversely affected by unstable market and economic conditions and potential macroeconomic effects, such as inflation, new or increased tariffs and higher interest rates, which may negatively impact our business, financial condition and share price.
For example, our ongoing Phase I/II clinical trial of AMT-130 is designed as an open-label trial following a 12-month core study period during which certain patients received a sham surgical procedure. Certain of these patients crossed over to treatment with AMT-130 and are now subject to long-term, unblinded follow-up monitoring for a period of five years.
For example, our ongoing Phase I/II clinical trial of AMT-130 is designed as an open-label trial following a 12-month core study period during which certain patients received a sham surgical procedure. Certain of these patients crossed over to an arm allowing for treatment with AMT-130 and are now subject to long-term, unblinded follow-up monitoring for a period of five years.
With respect to interim and preliminary data, the results and related findings and conclusions are subject to change following a more comprehensive review of the data, the particular study, or trial.
Additionally, with respect to interim and preliminary data, the results and related findings and conclusions are subject to change following a more comprehensive review of the data, the particular study, or trial.
This could reduce the ultimate demand for our product candidates or put pressure on our product pricing. Furthermore, there has been increased interest by third-party payors and governmental authorities in reference pricing systems and publication of discounts and list prices. Prescription drugs and biological products that are in violation of these requirements will be included on a public list.
This could reduce the ultimate demand for our product candidates or put pressure on our product pricing. Furthermore, there has been increased interest by third-party payers and governmental authorities in reference pricing systems and publication of discounts and list prices. Prescription drugs and biological products that are in violation of these requirements will be included on a public list.
To the extent that our employees, consultants, or contractors use technology or know-how owned by third parties in their work for us, disputes may arise between us and those third parties as to the rights in related inventions. 67 ​ Table of Contents Adequate remedies may not exist in the event of unauthorized use or disclosure of our confidential information including a breach of our confidentiality agreements.
To the extent that our employees, consultants, or contractors use technology or know-how owned by third parties in their work for us, disputes may arise between us and those third parties as to the rights in related inventions. ​ ​ ​ 71 ​ Table of Contents Adequate remedies may not exist in the event of unauthorized use or disclosure of our confidential information including a breach of our confidentiality agreements.
If an event of default occurs and the lender accelerates the amounts due, we may not be able to make accelerated payments, and the lender could seek to enforce security interests in the collateral securing such indebtedness, which includes substantially all our assets. Our 2024 Amended Facility bears a variable interest rate with a fixed floor. The U.S.
If an event of default occurs and the lender accelerates the amounts due, we may not be able to make accelerated payments, and the lender could seek to enforce security interests in the collateral securing such indebtedness, which includes substantially all our assets. Our 2025 Amended Facility bears a variable interest rate with a fixed floor. The U.S.
Risks Related to Our Business and the Development of Our Product Candidates We are dependent on the success of our lead product candidate, AMT-130, for the treatment of Huntington’s disease.
Risks Related to Our Business and the Development of Our Clinical Product Candidates We are dependent on the success of our lead clinical product candidate, AMT-130, for the treatment of Huntington’s disease.
Patients may also be reluctant to enroll in clinical trials for gene therapy candidates where other therapeutic alternatives are available due uncertainty about the safety or effectiveness gene therapies and the possibility that treatment with one gene therapy could preclude future gene therapy treatments due to the formation of antibodies following and in response to the treatment, or other unknown factors associated with novel therapeutics.
Patients may also be reluctant to enroll in clinical trials for gene therapy candidates where other therapeutic alternatives are available due to uncertainty regarding the safety or effectiveness of gene therapies and the possibility that treatment with one gene therapy could preclude future gene therapy treatments due to the formation of antibodies following and in response to the treatment, or other unknown factors associated with novel therapeutics.
Our reporting and compliance obligations may place a significant strain on our management, operational and financial resources, and systems for the foreseeable future. 80 ​ Table of Contents Our internal computer systems, or those of our collaborators, third-party vendors, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business and development programs.
Our reporting and compliance obligations may place a significant strain on our management, operational and financial resources, and systems for the foreseeable future. ​ ​ ​ ​ 86 ​ Table of Contents Our internal computer systems, or those of our collaborators, third-party vendors, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business and development programs.
Legally mandated price controls on payment amounts by third-party payors or other restrictions could seriously harm our business. In addition, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug healthcare programs.
Legally mandated price controls on payment amounts by third-party payers or other restrictions could seriously harm our business. In addition, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug healthcare programs.
If we are unable to meet these regulatory requirements, we may be delayed or not be able to obtain product approval. Certain of our product candidates require medical devices for administration, such as AMT-130 and AMT-260, each of which requires a stereotactic, magnetic resonance imaging guided catheter.
If we are unable to meet these regulatory requirements, we may be delayed or not be able to obtain product approval. Certain of our product candidates require medical devices for administration, such as AMT-130 and AMT-260, each of which requires a stereotactic, magnetic resonance imaging guided cannula.
The FDA, EMA, and other regulatory authorities will likely continue to revise and further update their approaches to gene therapies in the coming years.
The FDA, EMA, MHRA and other regulatory authorities will likely continue to revise and further update their approaches to gene therapies in the coming years.
If we or any of our third-party service providers fail to comply with applicable GCPs or other regulatory requirements, we or they may be subject to enforcement or other legal actions, the data generated in our trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional studies.
If we or any of our third-party service providers fail to comply with applicable cGCPs or other regulatory requirements, we or they may be subject to enforcement or other legal actions, the data generated in our trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional studies.
A U.S. holder may be able to make certain tax elections that would lessen the adverse impact of PFIC status; however, in order to make certain of such elections the U.S. holder will usually have to have been provided information about the company by us, and we do not intend to provide such information.
A U.S. holder may be eligible to make certain tax elections that would lessen the adverse impact of PFIC status; however, in order to make certain of such elections the U.S. holder will usually have to have been provided information about the company by us, and we do not intend to provide such information.
Interim data from our clinical trials, including the AMT-130 trial, and our analyses of that data are subject to the risk that one or more of our interim conclusions may materially change as more patient data become available and as regulatory interactions focused on statistical analysis of the clinical data progress, among other factors.
Topline or interim data from our clinical trials, including the AMT-130 trials, and our analyses of that data are subject to the risk that one or more of our conclusions may materially change as more patient data become available, and as regulatory interactions focused on statistical analysis of the clinical data progress, among other factors.
This restructuring, inclusive of the sale of our Lexington facility and associated employee transitions to Genezen, involved the elimination of approximately 65% of our global workforce, or approximately 300 roles across the company. We may encounter challenges in the execution of these and any future restructuring efforts, and these challenges could impact our financial results.
The 2024 restructuring, inclusive of the sale of our Lexington facility and associated employee transitions to Genezen, involved the elimination of approximately 65% of our global workforce, or approximately 300 roles across the company. We may encounter challenges in the execution of these and any future restructuring efforts, and these challenges could impact our financial results.
If we were required to, or if we chose to, discontinue development of AMT-130 or any other current or future product candidates, or if any of them were to fail to receive regulatory approval or achieve sufficient market acceptance, we could be prevented from or significantly delayed in achieving profitability and our business would be adversely affected. ​ 40 ​ Table of Contents We have encountered and may encounter future delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates.
If we were required, or if we chose, to discontinue development of AMT-130 or any other current or future product candidate, or if any of them were to fail to receive regulatory approval or achieve sufficient market acceptance, we could be prevented from or significantly delayed in achieving profitability and our business would be adversely affected. ​ ​ ​ ​ ​ 41 ​ Table of Contents We have encountered and may encounter future delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates.
By example, the EU has proposed exclusivity changes, in the form of draft legislation, that would effectively shorten the periods of EU orphan market exclusivity and data exclusivity. 52 ​ Table of Contents If we do not obtain or maintain periods of market exclusivity, we may face competition sooner than otherwise anticipated.
By example, the EU has proposed exclusivity changes, in the form of draft legislation, that would effectively shorten the periods of EU orphan market exclusivity and data exclusivity. ​ 55 ​ Table of Contents If we do not obtain or maintain periods of market exclusivity, we may face competition sooner than otherwise anticipated.
Events that may prevent successful or timely completion of clinical development, as well as product candidate approval, include, but are not limited to: ● occurrence of serious adverse events associated with a product candidate that are viewed to outweigh its potential benefits; ● insufficient number of patients treated with the product candidate or an insufficient study period for assessing the effectiveness of the product candidate; ● failures or delays in reaching agreement with regulatory agencies on study design, particularly with respect to our novel gene therapies for which regulatory pathways remain untested; ● failures or delays in hiring sufficient personnel with the requisite expertise to execute multiple clinical programs simultaneously; ● failures or delays in reaching agreement on acceptable terms with clinical research organizations (“CROs”) and clinical trial sites; ● failures or delays in identifying and recruiting patients in our clinical studies; ● delays in receiving regulatory authorization to conduct our clinical trials or a regulatory authority decision that the clinical trial should not proceed; ● failures or delays in obtaining or failure to obtain required IRB and IBC approval at each clinical trial site; ● requirements of regulatory authorities, IRBs, or IBCs to modify a study in such a way that it makes the study impracticable to conduct; ● regulatory authority requirements to perform additional or unanticipated clinical trials or testing; ● changes in standards of care which may necessitate the modification of our clinical trials or the conduct of new trials; ● regulatory authority refusal to accept data from foreign clinical study sites; ● disagreements with regulatory authorities regarding our study design, including endpoints, our chosen indication, our chosen bases for comparison as it relates to measurements of clinical efficacy, our interpretation and statistical analyses of data collected from preclinical studies and clinical trials; ● recommendations from DSMBs to discontinue, pause, or modify the trial; ● imposition of a clinical hold by regulatory agencies after an inspection of our clinical trial operations or trial sites; ● suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate; 41 ​ Table of Contents ● failure by CROs, other third parties or us to adhere to clinical trial requirements or otherwise properly manage the clinical trial process, including meeting applicable timelines, properly documenting case files, including the retention of proper case files, and properly monitoring and auditing clinical sites; ● failure of sites or clinical investigators to perform in accordance with Good Clinical Practice or applicable regulatory guidelines in other countries; ● failure of patients to abide by clinical trial requirements; ● delays or deviations in the testing, validation, manufacturing, and delivery of our product candidates to the clinical sites; ● delays in having patients complete participation in a study or return for post-treatment follow-up; ● clinical trial sites or patients dropping out of a study; ● the number of patients required for clinical trials of our product candidates being larger than we anticipate; ● clinical trials producing negative or inconclusive results, or our studies failing to reach the necessary level of statistical significance, requiring that we conduct additional clinical trials or abandon development programs; ● interruptions in manufacturing clinical supply of our product candidates or issues with product candidates failing to meet the necessary quality requirements; ● unanticipated clinical trial costs or insufficient funding, including paying substantial application user fees; ● emergence of new information about or impacting our product candidates or the field of gene therapy; ● determinations that there are issues with our third-party manufacturers or their facilities or processes; or ● changes in regulatory requirements and guidance, as well as new, revised, postponed, or frozen regulatory requirements (such as the EU Clinical Trials Regulation), that require amending or submitting new clinical protocols, undertaking additional new tests or analyses, or submitting new types or amounts of clinical data.
Events that may prevent successful or timely completion of clinical development, as well as clinical product candidate approval, include, but are not limited to: ● occurrence of serious adverse events associated with a product candidate that are viewed to outweigh its potential benefits; ● insufficient number of patients treated with the product candidate or an insufficient study period for assessing the effectiveness of the product candidate; ● failures or delays in reaching agreement with regulatory agencies on study design, particularly with respect to our novel gene therapies for which regulatory pathways remain untested; ● failures or delays in hiring sufficient personnel with the requisite expertise to execute multiple clinical programs simultaneously; ● failures or delays in reaching agreement on acceptable terms with clinical research organizations (“CROs”) and clinical trial sites; ● failures or delays in identifying, recruiting and enrolling patients in our clinical studies; ● delays in receiving regulatory authorization to conduct our clinical trials or a regulatory authority decision that the clinical trial should not proceed; ● failures or delays in obtaining or failure to obtain required IRB or Ethics Committee and IBC approval at each clinical trial site; 42 ​ Table of Contents ● requirements of regulatory authorities, IRBs, ECs, or IBCs to modify a study in such a way that it makes the study impracticable to conduct; ● regulatory authority requirements to perform additional or unanticipated clinical trials or testing; ● changes in standards of care which may necessitate the modification of our clinical trials or the conduct of new trials; ● regulatory authority refusal to accept data from foreign clinical study sites; ● disagreements with regulatory authorities regarding our study design, including endpoints, our chosen indication, our chosen bases for comparison as it relates to measurements of clinical efficacy, our interpretation and statistical analyses of data collected from preclinical studies and clinical trials; ● recommendations from Data and Safety Monitoring Boards (“DSMBs”) to discontinue, pause, or modify the trial; ● imposition of a clinical hold by regulatory agencies after an inspection of our clinical trial operations or trial sites; ● suspension or termination of clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate; ● failure by CROs, other third parties or us to adhere to clinical trial requirements or otherwise properly manage the clinical trial process, including meeting applicable timelines, properly documenting case files, including the retention of proper case files, and properly monitoring and auditing clinical sites; ● failure of sites or clinical investigators to perform in accordance with Good Clinical Practice or applicable regulatory guidelines in other countries; ● failure of patients to abide by clinical trial requirements; ● delays or deviations in the testing, validation, manufacturing, and delivery of our product candidates to the clinical sites; ● delays in having patients complete participation in a study or return for post-treatment follow-up; ● clinical trial sites or patients dropping out of a study; ● the number of patients required for clinical trials of our product candidates being larger than we anticipate; ● clinical trials producing negative or inconclusive results, or our studies failing to reach the necessary level of statistical significance, requiring that we conduct additional clinical trials or abandon development programs; ● interruptions in manufacturing clinical supply of our product candidates or issues with product candidates failing to meet the necessary quality requirements; ● unanticipated clinical trial costs or insufficient funding, including paying substantial application user fees; ● emergence of new information about or impacting our product candidates or the field of gene therapy; ● determinations that there are issues with our third-party manufacturers or their facilities or processes; or 43 ​ Table of Contents ● changes in regulatory requirements and guidance, including due to changes in policies or changes based on new or additional data or information, as well as new, revised, postponed, or frozen regulatory requirements, that require amending or submitting new clinical protocols, undertaking additional new tests or analyses, or submitting new types or amounts of clinical data.
We expect to finance our operations in 2025 primarily from our existing cash resources. We have devoted substantially all our financial resources and efforts to date to research and development of our products and product candidates, including the conduct of preclinical studies and clinical trials and related manufacturing requirements.
We expect to finance our operations in 2026 primarily from our existing cash resources. We have devoted substantially all our financial resources and efforts to date to research and development of our products and product candidates, including the conduct of preclinical studies and clinical trials and related manufacturing requirements.
For instance, our third-party service providers are not our employees, and except for remedies available to us under our agreements with such third parties we cannot control whether or not they devote sufficient time and resources to our ongoing clinical, non-clinical, and preclinical programs.
For instance, our third-party service providers are not our employees, and except for remedies available to us under our agreements with such third parties we cannot control whether or not they devote sufficient time and resources to our ongoing clinical, nonclinical, and preclinical programs.
Should Genezen encounter a manufacturing issues or if Genezen is unable to provide a sufficient supply of HEMGENIX® consistent with agreed-upon forecasting mechanisms, we may be unable to fulfil our contractual commitments to CSL Behring and may, thus, face contractual liabilities.
Should Genezen encounter a manufacturing issue or if Genezen is unable to provide a sufficient supply of HEMGENIX consistent with agreed-upon forecasting mechanisms, we may be unable to fulfil our contractual commitments to CSL Behring and may, thus, face contractual liabilities.
Moreover, the FDA and comparable foreign regulatory authorities require us to comply with GCPs for conducting, recording, and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity, and confidentiality of trial participants are protected.
Moreover, the FDA and comparable foreign regulatory authorities require us to comply with cGCPs for conducting, recording, and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity, and confidentiality of trial participants are protected.
There can be no assurance that we will be able to achieve all of our intended goals with respect to such strategies within the anticipated timeframes, if at all, or fully realize the expected benefits of any such transactions or arrangements. 70 ​ Table of Contents Divestitures (including the Lexington Transaction), product rationalizations or asset sales could result in asset impairments, or reductions to the size or scope of our business, our market share in particular markets or our opportunities and ability to compete with respect to certain markets, therapeutic areas or products.
There can be no assurance that we will be able to achieve all of our intended goals with respect to such strategies within the anticipated timeframes, if at all, or fully realize the expected benefits of any such transactions or arrangements. ​ ​ ​ 74 ​ Table of Contents Divestitures (such as the Lexington Transaction), product rationalizations or asset sales could result in asset impairments, or reductions to the size or scope of our business, our market share in particular markets or our opportunities and ability to compete with respect to certain markets, therapeutic areas or products.
There may be future changes in legal and regulatory requirements that may materially impact our results of operations. Future changes in legal and regulatory requirements may introduce new risks into our operations and future prospects, which we are not able to currently anticipate.
There may be future changes in legal and regulatory requirements or standards that may materially impact our results of operations. Future changes in legal and regulatory requirements may introduce new risks into our operations and future prospects, which we are not able to currently anticipate.
The addition of a new manufacturer may also require FDA, EMA, EU, and other regulatory authority approvals, which we may not be able to obtain. Our use of viruses, chemicals and other potentially hazardous materials requires us and our contract manufacturers to comply with regulatory requirements and exposes us to significant potential liabilities.
The addition of a new manufacturer may also require FDA, EMA, EU member state, MHRA and other regulatory authority approvals, which we may not be able to obtain. Our use of viruses, chemicals and other potentially hazardous materials requires us and our contract manufacturers to comply with regulatory requirements and exposes us to significant potential liabilities.
The Lexington Facility is and will continue to be subject to ongoing regulation and periodic inspection by the FDA, EU member state, and other regulatory bodies to ensure compliance with cGMP and other requirements.
The Lexington Facility and any other manufacturing facility is and will continue to be subject to ongoing regulation and periodic inspection by the FDA, EU member state, and other regulatory bodies to ensure compliance with cGMP and other requirements.
From time to time, we publicly disclose interim, preliminary or other data from preclinical studies and clinical trials, which are based on a preliminary and sometimes post hoc analysis of such data.
From time to time, we publicly disclose interim, preliminary, topline or other data from preclinical studies and clinical trials. Interim and preliminary data are based on a preliminary and sometimes post hoc analysis of such data.
We may leverage certain specialized regulatory pathways and designations, such as the FDA’s accelerated approval pathway and RMAT designation, to develop our product candidates or to seek licensure.
We may leverage certain specialized regulatory pathways and designations, such as the FDA’s accelerated approval pathway, RMAT designation and Breakthrough Therapy designation, to develop our product candidates or to seek licensure.
If we fail to obtain and sustain an adequate level of coverage and reimbursement for our products by third party payers, our ability to market and sell our products could be adversely affected and our business could be harmed. 54 ​ Table of Contents Due to the generally limited addressable market for our target orphan indications and the potential for our therapies to offer therapeutic benefit in a single administration, we face uncertainty related to our product candidates.
If we fail to obtain and sustain an adequate level of coverage and reimbursement for our products by third-party payers, our ability to market and sell our products could be adversely affected and our business could be harmed. 57 ​ Table of Contents Due to the generally limited addressable market for our target indications and the potential for our therapies to offer therapeutic benefit in a single administration, we face uncertainty related to pricing and reimbursement of our product candidates.
We expect to continue to rely on third parties, such as CROs, clinical data management organizations, medical and scientific institutions, and clinical and preclinical investigators, to conduct our preclinical studies and clinical trials. 62 ​ Table of Contents While we have agreements governing the activities of such third parties, we have limited influence and control over their actual performance and activities.
We expect to continue to rely on third parties, such as CROs, clinical data management organizations, medical and scientific institutions, and clinical and preclinical investigators, to conduct our preclinical studies and clinical trials. While we have agreements governing the activities of such third parties, we have limited influence and control over their actual performance and activities.
We have recently and historically pursued various strategic initiatives, transactions and business arrangements, including the July 2024 Lexington Transaction and the July 2021 acquisition of uniQure France and its lead program (AMT-260). We may, from time to time, enter into strategic transactions consistent with our business development and financial objectives.
We have historically pursued various strategic initiatives, transactions and business arrangements, including the July 2024 Lexington Transaction and the July 2021 acquisition of uniQure France (the “uniQure France Acquisition”) and its lead program (AMT-260). We may, from time to time, enter into strategic transactions consistent with our business development and financial objectives.
In addition, the information we choose to publicly disclose regarding a particular study or clinical trial may be top-line results based on what is typically extensive information, and others may not agree with what we determine is the material or otherwise appropriate information to include in our public disclosures.
In addition, the information we choose to publicly disclose regarding a particular study or clinical trial may be topline results based on what is typically extensive information, and others may not agree with what we determine is the material or otherwise appropriate information to include in our public disclosures.
Our third-party service providers may also have relationships with other entities, some of which may be our competitors, for whom they may also be conducting trials or other therapeutic development activities that could harm our competitive position. Our reliance on these third parties for development activities reduces our control over these activities.
Our third-party service providers may also have relationships with other entities, some of which may be our competitors, for whom they may also be conducting trials or other therapeutic development activities that could harm our competitive position. ​ ​ ​ ​ 66 ​ Table of Contents Our reliance on these third parties for development activities reduces our control over these activities.
Any collaboration we enter into may pose risks, including the following: ● collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations; ● we may have limited or no control over the design or conduct of clinical trials sponsored by collaborators; ● we may be hampered from entering into collaboration arrangements if we are unable to obtain consent from our licensors to enter into sublicensing arrangements of technology we have in-licensed; ● if any collaborator does not conduct the clinical trials they sponsor in accordance with regulatory requirements or stated protocols, we will not be able to rely on the data produced in such trials in our further development efforts; ● collaborators may not perform their obligations as expected; ● collaborators may also have relationships with other entities, some of which may be our competitors; ● collaborators may not pursue development and commercialization of any product candidates or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; ● collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial, or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; ● collaborators could develop, independently or with third parties, products that compete directly or indirectly with our products or product candidates, if, for instance, the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; ● our collaboration arrangements may impose restrictions on our ability to undertake other development efforts that may appear to be attractive to us; ● product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; ● a collaborator with marketing and distribution rights that achieves regulatory approval may not commit sufficient resources to the marketing and distribution of such product or products; ● disagreements with collaborators, including over proprietary rights, contract interpretation or the preferred course of development, could cause delays or termination of the research, development or commercialization of product candidates, lead to additional responsibilities for us, delay or impede reimbursement of certain expenses or result in litigation or arbitration, any of which would be time-consuming and expensive; ● collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our rights or expose us to potential litigation; ● collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and ● collaborations may in some cases be terminated for the convenience of the collaborator and, if terminated, we could be required to expend additional funds to pursue further development or commercialization of the applicable product or product candidates. ​ If any collaboration does not result in the successful research, development and commercialization of products or if a collaborator were to terminate an agreement with us, we may not receive future research funding or milestone or royalty payments under that collaboration, and we may lose access to important technologies and capabilities from the collaboration.
Any collaboration we enter into may pose risks, including the following: ● collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations; ● we may have limited or no control over the design or conduct of clinical trials sponsored by collaborators; ● we may be hampered from entering into collaboration arrangements if we are unable to obtain consent from our licensors to enter into sublicensing arrangements of technology we have in-licensed; ● if any collaborator does not conduct the clinical trials they sponsor in accordance with regulatory requirements or stated protocols, we will not be able to rely on the data produced in such trials in our further development efforts; 67 ​ Table of Contents ● collaborators may not perform their obligations as expected; ● collaborators may also have relationships with other entities, some of which may be our competitors; ● collaborators may not pursue development and commercialization of any product candidates or may elect not to continue or renew development or commercialization programs based on clinical trial results, changes in the collaborators’ strategic focus or available funding, or external factors, such as an acquisition, that divert resources or create competing priorities; ● collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial, or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; ● collaborators could develop, independently or with third parties, products that compete directly or indirectly with our products or product candidates, if, for instance, the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours; ● our collaboration arrangements may impose restrictions on our ability to undertake other development efforts that may appear to be attractive to us; ● product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own product candidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; ● a collaborator with marketing and distribution rights that achieves regulatory approval may not commit sufficient resources to the marketing and distribution of such product or products; ● disagreements with collaborators, including over proprietary rights, contract interpretation or the preferred course of development, could cause delays or termination of the research, development or commercialization of product candidates, lead to additional responsibilities for us, delay or impede reimbursement of certain expenses or result in litigation or arbitration, any of which would be time-consuming and expensive; ● collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our rights or expose us to potential litigation; ● collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and, ● collaborations may in some cases be terminated for the convenience of the collaborator and, if terminated, we could be required to expend additional funds to pursue further development or commercialization of the applicable product or product candidates.
For example: ● others may be able to make gene therapy products that are similar to our product candidates or utilize similar gene therapy technology but that are not covered by the claims of the patents that we own or have licensed; ● we or our licensors or future collaborators might not have been the first to make the inventions covered issued patents or pending patent applications that we own or have licensed; ● we or our licensors or future collaborators might not have been the first to file patent applications covering certain of our inventions; ● others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights; ● it is possible that our pending patent applications will not lead to issued patents; ● issued patents that we own or have licensed may be held invalid or unenforceable, as a result of legal challenges by our competitors; ● our competitors might conduct activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; ● we may not develop additional proprietary technologies that are patentable; and ● the patents of others may have an adverse effect on our business. 68 ​ Table of Contents The occurrence of any of these events could seriously harm our business.
For example: ​ ● others may be able to make gene therapy products that are similar to our product candidates or utilize similar gene therapy technology but that are not covered by the claims of the patents that we own or have licensed; ● we or our licensors or future collaborators might not have been the first to make the inventions covered issued patents or pending patent applications that we own or have licensed; ● we or our licensors or future collaborators might not have been the first to file patent applications covering certain of our inventions; ● others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our intellectual property rights; ● it is possible that our pending patent applications will not lead to issued patents; ● issued patents that we own or have licensed may be held invalid or unenforceable, as a result of legal challenges by our competitors; 72 ​ Table of Contents ● our competitors might conduct activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major commercial markets; ● we may not develop additional proprietary technologies that are patentable; and, ● the patents of others may have an adverse effect on our business.
For example, biologic product sponsors may be eligible for twelve years of exclusivity from the date of approval, seven years of exclusivity for drugs that are designated to be orphan drugs, and/or a six-month period of exclusivity added to any existing exclusivity period for the submission of FDA requested pediatric data.
For example, in the U.S., biologic product sponsors may be eligible for twelve years of exclusivity from the date of approval, seven years of exclusivity for drugs that are designated to be orphan drugs, and/or a six-month period of exclusivity added to any existing exclusivity period for the submission of FDA requested pediatric data.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

141 edited+54 added−70 removed45 unchanged
Biggest changeNet Cash used in operating activities ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 2022 ​ ​ ​ (in thousands) Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ (239,556) ​ $ (308,478) ​ $ (126,789) Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ ​ ​ ​ Depreciation, amortization and impairment ​ ​ 12,641 ​ ​ 11,900 ​ ​ 8,537 Amortization of discount on investment securities ​ ​ (10,901) ​ ​ (10,917) ​ ​ — Share-based compensation expense ​ ​ 22,258 ​ ​ 35,093 ​ ​ 34,204 Royalty financing agreement interest expense, net of interest paid ​ ​ 44,203 ​ ​ 25,616 ​ ​ — Deferred tax expense / (income) ​ ​ 2,429 ​ ​ 1,921 ​ ​ (1,470) Change in fair value of contingent consideration and derivative financial instrument, net ​ ​ (1,817) ​ ​ 15,895 ​ ​ 4,320 Unrealized foreign exchange losses / (gains), net ​ ​ 15,415 ​ ​ (2,206) ​ ​ (22,083) Other items, net ​ ​ (3,848) ​ ​ 4,721 ​ ​ 1,605 Changes in operating assets and liabilities: ​ ​ ​ ​ ​ ​ ​ ​ ​ Accounts receivable, prepaid expenses, and other current assets and receivables ​ ​ (2,243) ​ ​ (1,323) ​ ​ (4,083) Contract asset related to CSL Behring milestone payments ​ ​ - ​ ​ 100,000 ​ ​ (45,000) Inventories ​ ​ 2,421 ​ ​ (6,740) ​ ​ (6,924) Accounts payable ​ ​ 1,520 ​ ​ (4,169) ​ ​ 9,238 Accrued expenses, other liabilities, and operating leases ​ ​ (5,642) ​ ​ (5,328) ​ ​ 3,385 Contingent consideration milestone payment ​ ​ (19,608) ​ ​ (1,914) ​ ​ — Net cash used in operating activities ​ $ (182,728) ​ $ (145,929) ​ $ (145,060) ​ Net cash used in operating activities was $182.7 million for the year ended December 31, 2024, and consisted of a net loss of $239.6 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $12.6 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $22.3 million, $44.2 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $2.4 million, $1.8 million change in the fair value of contingent consideration and unrealized foreign exchange losses of $15.4 million.
Biggest changeIf we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, which could have a material adverse effect on our business, financial conditions, results of operations and cash flows. ​ ​ ​ 108 ​ Table of Contents Net Cash used in operating activities ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ 2024 ​ ​ 2023 ​ ​ ​ (in thousands) Cash flows from operating activities ​ ​ ​ ​ ​ ​ ​ ​ ​ Net loss ​ $ (198,971) ​ $ (239,556) ​ $ (308,478) Adjustments to reconcile net loss to net cash used in operating activities: ​ ​ ​ ​ ​ ​ ​ ​ ​ Depreciation, amortization and impairment ​ ​ 14,759 ​ ​ 12,641 ​ ​ 11,900 Amortization of discount on investment securities ​ ​ (10,463) ​ ​ (10,901) ​ ​ (10,917) Share-based compensation expense ​ ​ 17,679 ​ ​ 22,258 ​ ​ 35,093 Royalty financing agreement interest expense, net of interest paid ​ ​ 38,841 ​ ​ 44,203 ​ ​ 25,616 Deferred tax expense ​ ​ 1,588 ​ ​ 2,429 ​ ​ 1,921 Change in fair value of contingent consideration ​ ​ 6,247 ​ ​ (1,817) ​ ​ 15,895 Changes in fair value of liability related to pre-funded warrants ​ ​ (12,405) ​ ​ — ​ ​ — Unrealized foreign exchange (gains) / losses, net ​ ​ (23,019) ​ ​ 15,415 ​ ​ (2,206) Other items, net ​ ​ (5,429) ​ ​ (3,848) ​ ​ 4,721 Accounts receivable, prepaid expenses, and other current assets and receivables ​ ​ (8,597) ​ ​ (2,243) ​ ​ (1,323) Accounts payable ​ ​ (2,667) ​ ​ 1,520 ​ ​ (4,169) Accrued expenses, other liabilities, and operating leases ​ ​ 4,474 ​ ​ (5,642) ​ ​ (5,328) Contingent consideration milestone payments ​ ​ — ​ ​ (19,608) ​ ​ (1,914) Inventories ​ ​ — ​ ​ 2,421 ​ ​ (6,740) Contract asset related to CSL Behring milestone payment ​ ​ — ​ ​ — ​ ​ 100,000 Net cash used in operating activities ​ $ (177,963) ​ $ (182,728) ​ $ (145,929) ​ Net cash used in operating activities was $178.0 million for the year ended December 31, 2025 and consisted of a net loss of $199.0 million, adjusted for non-cash items including depreciation and amortization expense of $14.8 million, amortization of the discount on investment securities of $10.5 million, share-based compensation expense of $17.7 million, $38.8 million of interest expense net of interest paid related the Royalty Financing Agreement, a change in deferred taxes of $1.6 million, changes in the fair value of contingent consideration of $6.2 million, changes in the fair value of the liability related to pre-funded warrants of $12.4 million and unrealized foreign exchange gains of $23.0 million.
We incurred significant research and development costs related to manufacturing prior to the Closing of the Lexington Transaction and other enabling technologies that are applicable across all our programs. Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials.
We incurred significant research and development costs related to manufacturing prior to the closing of the Lexington Transaction and other enabling technologies that are applicable across all of our programs. Our R&D expenses may vary substantially from period to period based on the timing of our research and development activities, including regulatory submissions, and enrollment of patients in clinical trials.
Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX® to CSL and other milestones collected from CSL, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX®.
Between July 2021 and July 2023, we have collected $617.4 million from CSL Behring as a result of the sale of HEMGENIX to CSL Behring and other milestones collected from CSL Behring, and we are eligible to receive additional milestone payments, as well as royalties (to the extent not owed to settle the liability from the Royalty Financing Transaction) on net sales of HEMGENIX.
GAAP”) and unless otherwise indicated are presented in U.S. dollars. ​ Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements. Forward-looking statement are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied.
GAAP”) and unless otherwise indicated are presented in U.S. dollars. ​ Except for the historical information contained herein, the matters discussed in this MD&A may be deemed to be forward-looking statements. Forward-looking statements are only predictions based on management’s current views and assumptions and involve risks and uncertainties, and actual results could differ materially from those projected or implied.
This rule defines internal control over financial reporting as a process designed by, or under the supervision of, a company’s chief executive officer and chief financial officer and effected by our board of directors, management and other personnel, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of our assets that could have a material effect on the financial statements. ​ We assessed the effectiveness of our internal control over financial reporting as of December 31, 2024.
This rule defines internal control over financial reporting as a process designed by, or under the supervision of, a company’s chief executive officer and chief financial officer and effected by our board of directors, management and other personnel, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company; (2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company; and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of our assets that could have a material effect on the financial statements. ​ We assessed the effectiveness of our internal control over financial reporting as of December 31, 2025.
As a result of being an agent, we recognize corresponding costs related to the purchase of HEMGENIX® from Genezen net of income from the sales of HEMGENIX® to CSL Behring and income related to the release of liabilities associated with expected net losses in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.
As a result of our being an agent, we recognize corresponding costs related to the purchase of HEMGENIX from Genezen, net of income from the sales of HEMGENIX to CSL Behring, and income related to the release of liabilities associated with expected net losses, in Other expense within the Company’s Consolidated Statements of Operations and Comprehensive Loss.
Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the periods ended December 31, 2023 and 2024 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition. ● Preclinical research programs .
Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the periods ended December 31, 2025, 2024 and 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition; ● Preclinical research programs .
Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include: ● employee-related expenses, including salaries, benefits, travel and share-based compensation expense; ● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors; ● costs incurred to conduct consistency and comparability studies; ● costs incurred for the development and improvement of our manufacturing processes and methods; ● costs associated with research activities for enabling technology platforms; ● costs associated with the rendering of collaboration services; ● payments related to identifiable intangible assets without an alternative future use; ● payments to our licensors for milestones that have been achieved related to our product candidates; ● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and ● changes in the fair value of liabilities recorded in relation to our acquisition of uniQure France SAS. ​ ​ 94 ​ Table of Contents Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include: ​ ● AMT-130 (Huntington’s disease).
Our R&D expenses generally consist of costs incurred for the development of our target candidates, which include: ● employee-related expenses, including salaries, benefits, travel and share-based compensation expense; ● costs incurred for laboratory research, preclinical and nonclinical studies, clinical trials, statistical analysis and report writing, and regulatory compliance costs incurred with clinical research organizations and other third-party vendors; ● costs incurred to conduct consistency and comparability studies; ● costs incurred for the development and improvement of our manufacturing processes and methods; ● costs associated with research activities for enabling technology platforms; ● costs associated with the rendering of collaboration services; ● payments related to identifiable intangible assets without an alternative future use; ● payments to our licensors for milestones that have been achieved related to our product candidates; ● facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance, and other supplies; and, ● changes in the fair value of liabilities recorded in relation to the uniQure France Acquisition. ​ 98 ​ Table of Contents Our research and development expenses primarily consist of costs incurred for the research and development of our product candidates, which include: ● AMT-130 (Huntington’s disease).
Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million. There was an decrease in inventory balances of $2.4 million.
Net cash generated from operating activities also included unfavorable changes in operating assets and liabilities of $3.9 million. There was a net increase in accounts receivable, prepaid expenses, and other current assets and receivables of $2.2 million. There was a decrease in inventory balances of $2.4 million.
In the years ended December 31, 2024 and 2023, we incurred $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial in which enrolled the first patient in October 2024.
In the years ended December 31, 2025, 2024 and 2023, we incurred $6.3 million, $7.3 million and $6.0 million of expenses, respectively, to initiate a Phase I/II clinical trial, in which we enrolled the first patient in October 2024.
To defend against, detect and respond to cybersecurity incidents, we, among other things: have enabled regular monitoring of our environment by a combination of external partners and internal security tools, conduct regular employee trainings, monitor emerging laws and regulations related to data protection and information security and implement appropriate changes. ​ Consistent with our cybersecurity risk management policies and controls, we have prepared and implemented an incident response plan with several components, including: (i) engagement of a third party security operations center for regular vulnerability scanning and technical monitoring of our systems, (ii) detection and analysis of cybersecurity incidents that present risk of unauthorized access to company assets, including the escalation and triaging of incidents that present acute risks to our business, (iii) containment, eradication and data recovery, and (iv) post-incident analysis.
To defend against, detect and respond to cybersecurity incidents, we, among other things: have enabled regular monitoring of our environment by a combination of external partners and internal security tools, conduct regular employee trainings, monitor emerging laws and regulations related to data protection and information security and implement appropriate changes. 88 ​ Table of Contents Consistent with our cybersecurity risk management policies and controls, we have prepared and implemented an incident response plan with several components, including: (i) engagement of a third party security operations center for regular vulnerability scanning and technical monitoring of our systems, (ii) detection and analysis of cybersecurity incidents that present risk of unauthorized access to company assets, including the escalation and triaging of incidents that present acute risks to our business, (iii) containment, eradication and data recovery, and (iv) post-incident analysis.
The majority of the U.S. patent rights will expire in 2028 and the European patent rights (along with one U.S. patent) will expire in 2025. With respect to our collaboration with CSL Behring, we have agreed with St.
The majority of the U.S. patent rights will expire in 2028 and the European patent rights (along with one U.S. patent) expired in 2025. With respect to our collaboration with CSL Behring, we have agreed with St.
There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to a increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals.
There was a net decrease in accounts payable, accrued expenses, other liabilities, and operating leases of $4.1 million, primarily related to an increase of $1.5 million in accounts payable and a decrease of $5.6 million related to various accruals.
Cybersecurity risks related to our business are identified and addressed through a multi-faceted approach that consists of third-party assessments, testing of our information systems and information technology security.
Cybersecurity risks related to our business are identified and addressed through a multi-faceted approach that consists of periodic third-party assessments, testing of our information systems and information technology security.
In our management’s opinion, we have maintained effective internal control over financial reporting as of December 31, 2024, based on criteria established in the COSO 2013 framework. ​ Our independent registered public accounting firm, which has audited the consolidated financial statements included in this Annual Report on Form 10-K, has also issued an audit report on the effectiveness of our internal control over financial reporting as of December 31, 2024.
In our management’s opinion, we have maintained effective internal control over financial reporting as of December 31, 2025, based on criteria established in the COSO 2013 framework. ​ Our independent registered public accounting firm, which has audited the consolidated financial statements included in this Annual Report on Form 10-K, has also issued an audit report on the effectiveness of our internal control over financial reporting as of December 31, 2025.
The performance of our ordinary shares shown on the graph below is not necessarily indicative of the future performance of our ordinary shares. ​ 85 ​ Table of Contents This graph and related information are not “soliciting material,” is not deemed “filed” with the SEC and, except to the extent incorporated by reference, is not to be incorporated by reference into any of our filings under the Securities Act, or the U.S.
The performance of our ordinary shares shown on the graph below is not necessarily indicative of the future performance of our ordinary shares. 91 ​ Table of Contents This graph and related information are not “soliciting material,” is not deemed “filed” with the SEC and, except to the extent incorporated by reference, is not to be incorporated by reference into any of our filings under the Securities Act, or the U.S.
Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgement due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of: ● the scope, rate of progress and expense of our research and development activities; ● clinical trial protocols, speed of enrollment and resulting data; ● the effectiveness and safety of our product candidates; and ● the timing of regulatory approvals.
Estimating the nature, timing, or cost of the development of any of our product candidates involves considerable judgment due to numerous risks and uncertainties associated with developing gene therapies, including the uncertainty of: ● the scope, rate of progress and expense of our research and development activities; ● clinical trial protocols, speed of enrollment and resulting data; ● the effectiveness and safety of our product candidates; and, ● the timing of regulatory approvals.
This graph assumes an investment of $100 after market close on December 31, 2019 in each of our ordinary shares, the NASDAQ Composite Index, and the NASDAQ Biotechnology Index. Pursuant to the applicable SEC rules, all values assume reinvestment of the full amount of all dividends; however, no dividends have been declared on our ordinary shares to date.
This graph assumes an investment of $100 after market close on December 31, 2020 in each of our ordinary shares, the NASDAQ Composite Index, and the Nasdaq Biotechnology Index. Pursuant to the applicable SEC rules, all values assume reinvestment of the full amount of all dividends; however, no dividends have been declared on our ordinary shares to date.
In January 2025, we raised $70.1 million of net proceeds, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a price to the public of $17.00 per ordinary share.
Financing Public offerings In January 2025, we raised $70.1 million of net proceeds, after deducting underwriting discounts and commissions and other offering expenses payable by us, through a follow-on public offering of 4.4 million ordinary shares at a price to the public of $17.00 per ordinary share.
The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. The timing of achieving these milestones, as well as whether the milestone will be achieved at all, and consequently the timing of payments is generally uncertain. We expect these obligations will become payable between 2029 and 2033.
The commitments include payments related to post-acquisition services that we agreed to as part of the transaction. The timing of achieving these milestones, as well as whether the milestone will be achieved at all, and consequently the timing of payments is generally uncertain. We expect these obligations will become payable between 2028 and 2033.
We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations and fees related to business development and maintaining our patent and license portfolio.
We incurred expenses associated with operating as a public company, including expenses for personnel, legal, accounting and audit fees, board of directors’ costs, directors' and officers' liability insurance premiums, Nasdaq listing fees, expenses related to investor relations, expenses to maintain our patent and license portfolio, and fees related to business development.
Net cash used in operating activities also includes a payment for a contingent consideration milestone of $19.6 million. 103 ​ Table of Contents Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the May 2023 royalty financing agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.
Net cash used in operating activities also includes a payment for a contingent consideration payment of $19.6 million. 109 ​ Table of Contents Net cash used in operating activities was $145.9 million for the year ended December 31, 2023, and consisted of a net loss of $308.5 million adjusted for non-cash items, including depreciation, amortization and impairment expense of $11.9 million, amortization of the discount on investment securities of $10.9 million, share-based compensation expense of $35.1 million, $25.6 million of interest expense net of interest paid related to the Royalty Financing Agreement, a change in deferred taxes of $1.9 million, $15.9 million change in the fair value of contingent consideration and unrealized foreign exchange gains of $2.2 million.
Controls and Procedures . ​ Evaluation of Disclosure Controls and Procedures ​ Our management, with the participation of our chief executive officer (“CEO”, our principal executive officer) and chief financial officer (“CFO”, our principal financial officer), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act) as of December 31, 2024.
Controls and Procedures . ​ Evaluation of Disclosure Controls and Procedures ​ Our management, with the participation of our chief executive officer (“CEO”, our principal executive officer) and chief financial officer (“CFO”, our principal financial officer), evaluated the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act) as of December 31, 2025.
We recognized $3.2 million in net other expenses during the year ended December 31, 2024 (nil in 2023 and 2022) related to the purchase of HEMGENIX® from Genezen, net of income from the sales of HEMGENIX® to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.
We recognized $6.3 million and $3.2 million in net other expenses during the year ended December 31, 2025 and 2024 and nil in 2023, related to the purchase of HEMGENIX from Genezen, net of income from the sales of HEMGENIX to CSL Behring, amortization of the intangible asset for our favorable supply terms under the CSA and credits from the release of liabilities related to expected net losses associated with minimum purchase commitments under the CSA.
Securities Exchange Act of 1934, as amended (the “Exchange Act”), whether made before or after the date hereof and irrespective of any general incorporation language in any such filing. ​ Item 6. Reserved ​ ​ ​ ​ ​ ​ 86 ​ Table of Contents Item 7.
Securities Exchange Act of 1934, as amended (the “Exchange Act”), whether made before or after the date hereof and irrespective of any general incorporation language in any such filing. Item 6. Reserved ​ ​ ​ ​ 92 ​ Table of Contents Item 7.
Other income for the years ended December 31, 2024, 2023, and 2022 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility. We present expenses related to such income as other expense.
Other income for the years ended December 31, 2025, 2024, and 2023 also includes income from the subleasing of a portion of our Amsterdam facility and Lexington research and development facility. We present expenses related to such income as other expense.
This MD&A is provided as a supplement to, and should be read in conjunction with, our audited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors.” Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S.
This MD&A is provided as a supplement to, and should be read in conjunction with, our audited consolidated financial statements and the accompanying notes thereto and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors”. Our consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the U.S. (“U.S.
We incurred $1.3 million, $0.1 million and $1.3 million of such cost in the years ended December 31, 2024, 2023 and 2022, respectively. We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied.
We incurred $1.7 million, $1.3 million and $0.1 million of such cost in the years ended December 31, 2025, 2024 and 2023, respectively. We recognize collaboration revenues associated with services we provide to CSL Behring. Collaboration revenue is recognized when the performance obligations are satisfied.
Based on such evaluation, our CEO and CFO have concluded that as of December 31, 2024, our disclosure controls and procedures were effective. 108 ​ Table of Contents ​ Management’s Annual Report on Internal Control Over Financial Reporting ​ Our management is responsible for establishing and maintaining adequate internal control over financial reporting, as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act.
Based on such evaluation, our CEO and CFO have concluded that as of December 31, 2025, our disclosure controls and procedures were effective. 114 ​ Table of Contents Management’s Annual Report on Internal Control Over Financial Reporting ​ Our management is responsible for establishing and maintaining adequate internal control over financial reporting, as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act.
In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS following the dosing of the first patient in Phase I/II clinical trial for AMT-260.
In December 2024, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260.
We anticipate that we will retain all earnings, if any, to support operations and to finance the growth and development of our business for the foreseeable future. ​ Unregistered Sales of Equity Securities ​ During the period covered by this Annual Report on Form 10-K, we have not issued any securities that were not registered under the Securities Act of 1933, as amended (the “Securities Act”). ​ Issuer Purchases of Equity Securities ​ We did not make any purchases of our ordinary shares during the period covered by this Annual Report on Form 10-K. ​ Holders ​ As of February 24, 2025, there were approximately three holders of record of our ordinary shares.
We anticipate that we will retain all earnings, if any, to support operations and to finance the growth and development of our business for the foreseeable future. ​ Unregistered Sales of Equity Securities ​ During the period covered by this Annual Report on Form 10-K, we have not issued any securities that were not registered under the Securities Act of 1933, as amended (the “Securities Act”). ​ Issuer Purchases of Equity Securities ​ We did not make any purchases of our ordinary shares during the period covered by this Annual Report on Form 10-K. ​ Holders ​ As of February 26, 2026, there were approximately nine holders of record of our ordinary shares.
On an ongoing basis, we evaluate our assumptions, estimates and judgments, including those related to what we believe to be our critical accounting policies. Refer to Note 2 “Summary of significant accounting policies” for a summary of our significant accounting policies.
On an ongoing basis, we evaluate our assumptions, estimates and judgments, including those related to what we believe to be our critical accounting policies. Refer to Note 2 “ Summary of significant accounting policies ” for a summary of our significant accounting policies.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements due to error or fraud. ​ Changes in internal control over financial reporting During the fourth quarter of 2024, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting. ​ Item 9B.
Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements due to error or fraud. ​ Changes in internal control over financial reporting During the fourth quarter of 2025, there were no changes in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) that materially affected, or are reasonably likely to materially affect, our internal control over financial reporting. ​ ​ ​ ​ 115 ​ Table of Contents Item 9B.
Our cash and cash equivalents include bank balances, demand deposits and other short-term highly liquid investments (with maturities of less than three months at the time of purchase) that are readily convertible into a known amount of cash and are subject to an insignificant risk of fluctuation in value. Restricted cash includes deposits made in relation to facility leases.
Our cash and cash equivalents include bank balances, demand deposits and other short-term highly liquid investments (with maturities of less than three months at the time of purchase) that are readily convertible into a known amount of cash and are subject to an insignificant risk of fluctuation in value. ​ Restricted cash includes deposits made in relation to facility leases in Amsterdam, the Netherlands and Lexington, Massachusetts, and Basel, Switzerland.
The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses partially offset by decreases in contractual license expenses and consultant-related expenses.
The decrease in costs in 2025 of $4.9 million, compared to 2024, is primarily due to a decrease in information technology expenses and consultant-related expenses. The increase in costs in 2024 of $2.9 million, compared to 2023, is primarily a result of an increase in information technology expenses, partially offset by decreases in contractual license expenses and consultant-related expenses.
We incurred $17.1 million of cost of contract manufacturing revenues related to the manufacture of HEMGENIX® in the year ended December 31, 2024 and $13.6 million and $2.1 million of cost of contract manufacturing revenues, in the years ended December 31, 2023 and 2022 respectively.
We incurred nil cost of contract manufacturing revenues related to the manufacture of HEMGENIX in the year ended December 31, 2025 and $17.1 million and $13.6 million of cost of contract manufacturing revenues, in the years ended December 31, 2024 and 2023, respectively.
We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease; ● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ) We have incurred costs related to the acquisition in addition to costs incurred to initiate and conduct a Phase I/II clinical trial; ● AMT-061 (Hemophilia B) .
We have incurred costs related to the development of AMT-191 for the treatment of Fabry disease and costs to initiate and conduct a Phase I/II clinical trial; ● AMT-162 ( Amyotrophic Lateral Sclerosis caused by mutations in SOD1 ).
We are subject to certain covenants under the debt facility and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.
We are subject to certain covenants under the senior secured term loan facility with Hercules and may become subject to covenants under any future indebtedness that could limit our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends, which could adversely impact our ability to conduct our business.
We recognized contract manufacturing revenues related to contract manufacturing HEMGENIX ® for CSL Behring when earned upon sales of HEMGENIX ® to CSL Behring. We recognized $6.1 million, $10.8 million and $1.7 million contract manufacturing revenues in the years ended December 31, 2024, 2023 and 2022, respectively.
We recognized contract manufacturing revenues related to the contract manufacture of HEMGENIX for CSL Behring when earned upon sales of HEMGENIX to CSL Behring. We recognized nil, $6.1 million and $10.8 million of contract manufacturing revenues in the years ended December 31, 2025, 2024 and 2023, respectively.
Conversely, if the euro had strengthened 10% against the U.S. dollar with all other variables held constant, pre-tax loss for the year would have been $25.0 million lower (December 31, 2023: pre-tax loss $6.0 million lower), and other comprehensive income would have been $12.1 million lower (December 31, 2023: other comprehensive loss $2.6 million lower). ​ We strive to mitigate foreign exchange risk through holding sufficient funds in euro and dollars to finance budgeted cash flows for generally 18 months. ​ The sensitivity in other comprehensive income to fluctuations in exchange rates primarily relates to the translation of the net assets of our Dutch entities from their functional currency euro into our reporting currency U.S. dollar. ​ Price risk ​ The market prices for the provision of preclinical and clinical materials and services, as well as external contracted research, may vary over time. ​ The commercial prices of any of our products or product candidates are currently uncertain. ​ We are not exposed to commodity price risk. ​ We do not hold investments classified as available-for-sale or at fair value through profit or loss; therefore, we are not exposed to equity securities price risk. ​ Interest rate risk ​ Our interest rate risk arises from short- and long-term debt and investment securities. ​ In June 2013, we entered into the Hercules Agreement, which was last amended in July 2024, under which our borrowings bear interest at a variable rate with a fixed floor.
Conversely, if the euro had strengthened 10% against the U.S. dollar with all other variables held constant, the pre-tax loss for the year would have been $12.4 million lower (December 31, 2024: pre-tax loss $25.0 million lower), and other comprehensive loss would have been $1.9 million higher (December 31, 2024: other comprehensive loss $12.1 million lower). ​ We strive to mitigate foreign exchange risk through holding sufficient funds in euro and U.S. dollars, to finance budgeted cash flows for generally 18 months. ​ The sensitivity in other comprehensive income to fluctuations in exchange rates primarily relates to the translation of the net assets of our Dutch entities from their functional currency, the euro, into our reporting currency, the U.S. dollar. ​ Price risk ​ The market prices for the provision of preclinical and clinical materials and services, as well as external contracted research, may vary over time. ​ The commercial prices of any of our products or product candidates are currently uncertain. ​ We are not exposed to commodity price risk. ​ We do not have a material exposure to equity securities price risk, as we do not hold marketable equity securities. ​ Interest rate risk ​ Our interest rate risk arises from short- and long-term debt and investment securities. ​ In June 2013, we entered into a venture debt loan facility with Hercules, which was last amended in September 2025, under which our borrowings bear interest at a variable rate with a fixed floor.
We lease an additional approximately 12,000 square feet of space in the Amsterdam facility, which is subject to a lease that expires in December 2027. ​ We operate approximately 7,400 square feet of multi-use office space in Basel, Switzerland, which is subject to a lease that expires in 2026 and may be renewed for two subsequent terms of three years. ​ We believe that our facilities are adequate to meet current needs and that suitable alternative spaces will be available in the future on commercially reasonable terms. ​ Item 3.
We lease an additional approximately 12,000 square feet of space in the Amsterdam facility, which is subject to a lease that expires in December 2027. ​ We operate approximately 7,400 square feet of multi-use office space in Basel, Switzerland, which is subject to a lease that, as amended to date, expires in 2028 and may be renewed for a subsequent term of two years. ​ 89 ​ Table of Contents We believe that our facilities are adequate to meet current needs and that suitable additional or alternative spaces will be available in the future on commercially reasonable terms. ​ ​ Item 3.
Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 and 2022 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition. ​ Other research & development expenses ​ ● We incurred $49.8 million in employee and contractor expenses in the year ended December 31, 2024 compared to $74.6 million in 2023 and $64.9 million in 2022.
Direct research and development expenses related to clinical development and other regulatory activities and commercialization expenses incurred in the years ended December 31, 2023 are presented net of reimbursements due from CSL Behring and include settlement amounts from the transition. ​ Other research & development expenses ​ ● We incurred $35.3 million in employee and contractor expenses in the year ended December 31, 2025 compared to $49.8 million in 2024 and $74.5 million in 2023.
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidate. ​ 95 ​ Table of Contents Research and development expenses for the year ended December 31, 2024 were $143.8 million, compared to $214.9 million and $197.6 million for the years ended December 31, 2023 and 2022, respectively.
A change in the outcome of any of these variables with respect to our product candidates that we may develop could mean a significant change in the expenses and timing associated with the development of such product candidates. ​ 99 ​ Table of Contents Research and development expenses for the year ended December 31, 2025 were $140.7 million, compared to $143.8 million and $214.9 million for the years ended December 31, 2024 and 2023, respectively.
Funding requirements ​ Our future capital requirements will depend on many factors, including but not limited to: ● activities related to submission of a BLA for AMT-130 in Huntington’s disease; ● activities to prepare for the commercialization of AMT-130 in Huntington’s disease in the United States; ● investments to launch AMT-130 in Huntington’s disease in the United States including the amounts of revenue we generate following the launch; ● earnout payments we might owe the former shareholders of uniQure France SAS, which are subject to the achievement of specific development and regulatory milestones; ● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement; ● the scope, timing, results, and costs of our current and planned clinical trials; ● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements; ● the extent to which we acquire or in-license other businesses, products, product candidates or technologies; ● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates; ● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates; ● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies; ● the cost, timing and outcome of regulatory reviews associated with our product candidates; ● our ability to enter into collaboration arrangements in the future; and ● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims. ​ 105 ​ Table of Contents Item 7A.
Funding requirements ​ Our future capital requirements will depend on many factors, including but not limited to: ● Investments to develop AMT-130, AMT-191 and AMT-260 beyond completing the ongoing trials; ● activities to prepare for the potential commercialization of AMT-130 for Huntington’s disease, if we can align on the requirements for marketing authorization with the respective regulatory authorities; ● earnout payments we might owe the former shareholders of uniQure France, which are subject to the achievement of specific development and regulatory milestones; ● contractual milestone payments and royalties we might be owed in accordance with the CSL Behring Agreement; ● the scope, timing, results, and costs of our current and planned clinical trials; ● the scope, obligations and restrictions on our business related to our existing equity, debt or royalty monetization financings and underlying agreements; ● the extent to which we acquire or in-license other businesses, products, product candidates or technologies; ● the scope, timing, results and costs of preclinical development and laboratory testing of our additional product candidates; ● the need for additional resources and related recruitment costs to support the preclinical and clinical development of our product candidates; ● the need for any additional tests, studies, or trials beyond those originally anticipated to confirm the safety or efficacy of our product candidates and technologies; ● the cost, timing and outcome of regulatory reviews associated with our product candidates; ● our ability to enter into collaboration arrangements in the future; and ● the costs and timing of preparing, filing, expanding, acquiring, licensing, maintaining, enforcing, and prosecuting patents and patent applications, as well as defending any intellectual property-related claims. ​ 111 ​ Table of Contents Item 7A.
For a discussion of cybersecurity risks applicable to us, see Part I, Item 1A, Risk Factors , under the heading “Our internal computer systems, or those of our collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs” in this Annual Report on Form 10-K. ​ ​ 83 ​ Table of Contents Item 2.
For a discussion of cybersecurity risks applicable to us, see Part I, Item 1A, Risk Factors, under the heading “Our internal computer systems, or those of our collaborators, third-party vendors, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business and development programs” in this Annual Report on Form 10-K. ​ Item 2.
As of December 31, 2024 our remaining minimum purchase commitments amount to $12.5 million with $4.0 million to be paid within the next 12 months. ​ The table below summarizes our consolidated cash flow data for the years ended December 31: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 2022 ​ ​ (in thousands) Cash, cash equivalents and restricted cash at the beginning of the period ​ $ 244,544 ​ $ 231,173 ​ $ 559,353 Net cash used in operating activities ​ ​ (182,728) ​ ​ (145,929) ​ ​ (145,060) Net cash generated from / (used in) investing activities ​ ​ 162,968 ​ ​ (205,686) ​ ​ (182,734) Net cash (used in) / generated from financing activities ​ ​ (59,486) ​ ​ 362,721 ​ ​ 1,445 Foreign exchange impact ​ ​ (4,969) ​ ​ 2,265 ​ ​ (1,831) Cash, cash equivalents and restricted cash at the end of period ​ $ 160,329 ​ $ 244,544 ​ $ 231,173 ​ We had previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V. in 1998, with the exception of generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement.
As of December 31, 2025 our remaining minimum purchase commitments amount to $7.3 million with $4.3 million to be paid within the next 12 months. ​ The table below summarizes our consolidated cash flow data for the years ended December 31, 2025, 2024, and 2023 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 ​ ​ (in thousands) Cash, cash equivalents and restricted cash at the beginning of the period ​ $ 160,329 ​ $ 244,544 ​ $ 231,173 Net cash used in operating activities ​ ​ (177,963) ​ ​ (182,728) ​ ​ (145,929) Net cash (used in) / generated from investing activities ​ ​ (321,619) ​ ​ 162,968 ​ ​ (205,686) Net cash generated from / (used in) financing activities ​ ​ 415,398 ​ ​ (59,486) ​ ​ 362,721 Foreign exchange impact ​ ​ 5,657 ​ ​ (4,969) ​ ​ 2,265 Cash, cash equivalents and restricted cash at the end of period ​ $ 81,802 ​ $ 160,329 ​ $ 244,544 ​ We have previously incurred losses and cumulative negative cash flows from operations since our business was founded by our predecessor entity AMT Holding N.V. in 1998, with the exception of our generating income in 2021 after receiving the upfront payment upon Closing of the CSL Behring Agreement.
We are currently conducting the U.S. and European studies. We completed the enrollment of all 26 patients in the first two cohorts of our US study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.
We completed the enrollment of all 26 patients in the first two cohorts of our U.S. study in March 2022 and the enrollment of 13 patients in the two cohorts of our European study in June 2023.
We recognized nil income related to our equity stake in VectorY B.V. in the year ended December 31, 2024. In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V. following an October 2023 financing round.
In 2023, we recognized $0.8 million in other expense related to a reduction in the fair market value of our equity stake in VectorY B.V. following an October 2023 financing round.
The debt facility permits us to issue up to $500.0 million of convertible debt. 102 ​ Table of Contents To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all.
To the extent we need to finance our cash needs through equity offerings or debt financings, such financing may be subject to unfavorable terms including without limitation, the negotiation and execution of definitive documentation, as well as credit and debt market conditions, and we may not be able to obtain such financing on terms acceptable to us or at all.
As of December 31, 2024, the loan bore an interest rate of 12.2%. 106 ​ Table of Contents ​ As of December 31, 2024, if interest rates on borrowings had been 1.0% higher with all other variables held constant, pre-tax earnings for the year would have been $0.8 million lower (2023: $1.0 million lower; 2022: $1.0 million lower.) ​ We invest in government debt in accordance with our investment policy.
As of December 31, 2025, the loan bore a nominal interest rate of 9.45%. ​ ​ 112 ​ Table of Contents As of December 31, 2025, if interest rates on borrowings had been 1.0% higher with all other variables held constant, pre-tax earnings for the year would have been $0.5 million lower (2024: $0.8 million lower; 2023: $1.0 million lower.) ​ We invest in government debt in accordance with our investment policy.
Due to the high credit quality of our counterparties, we believe there is no material exposure to credit risk in our portfolio of investment securities. ​ Liquidity Risk ​ Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe that our cash and cash equivalents and investment securities will fund our operations through the second half of 2027.
Due to the high credit quality of our counterparties, we believe there is no material exposure to credit risk in our portfolio of investment securities. ​ Liquidity Risk ​ Based on our current operating plan, research and development plans and our timing expectations related to the progress of our programs, we believe our cash and cash equivalents, restricted cash, and investment securities will be sufficient to fund our projected operating expenses into the second half of 2029.
Commitments related to acquisition of uniQure France SAS (nominal amounts) In relation to our acquisition of uniQure France SAS, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractual milestones. The commitments include payments related to post-acquisition services that we agreed to as part of the transaction.
Commitments related to the uniQure France Acquisition (nominal amounts) In connection with the uniQure France Acquisition, we entered into commitments to make payments to the former shareholders upon the achievement of certain contractually defined milestones. The commitments include payments related to post-acquisition services that we agreed as part of the transaction.
For example, if we hold a security that was issued at a fixed interest rate at the then-prevailing rate and the prevailing interest rate later rises, the fair value of our investment will probably decline. ​ The duration of all of our investment securities held as of December 31, 2024, was between two to five months.
For example, if we hold a security that was issued at a fixed interest rate at the then-prevailing rate and the prevailing interest rate later rises, the fair value of our investment will probably decline. ​ All of our investment securities held as of December 31, 2025, had maturities ranging between one to nine months.
As of December 31, 2024, we had an accumulated deficit of $1,130.0 million. ​ Sources of liquidity ​ From our first institutional venture capital financing in 2006 through the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023.
As of December 31, 2025, we had an accumulated deficit of $1,328.9 million. 107 ​ Table of Contents Sources of liquidity ​ From our first institutional venture capital financing in 2006 through to the current period, we funded our operations primarily through private and public placements of equity securities, debt securities, payments from our collaboration partners, as well as $370.1 million from selling a portion of royalties due from our collaboration partner CSL Behring in 2023 in connection with the Royalty Financing Transaction.
Commitments related to licensors and financial advisors We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our License Sale to CSL Behring.
If and when due, up to 25% of the milestone payments can be settled with our ordinary shares. ​ ​ 106 ​ Table of Contents Commitments related to licensors and financial advisors We have obligations to make future payments to third parties that become due and payable on the achievement of certain development, regulatory and commercial milestones (such as the start of a clinical trial, filing of a BLA, approval by the FDA or product launch) or as a result of collecting payments related to our License Sale to CSL Behring.
On December 31, 2024, if the euro had weakened 10% against the U.S. dollar with all other variables held constant, pre-tax loss for the year would have been $25.0 million higher (December 31, 2023: pre-tax loss $6.0 million higher), and other comprehensive income would have been $11.0 million higher (December 31, 2023: other comprehensive loss $0.3 million higher).
On December 31, 2025, if the euro had weakened 10% against the U.S. dollar with all other variables held constant, the pre-tax loss for the year would have been $12.4 million higher (December 31, 2024: pre-tax loss $25.0 million higher), and other comprehensive loss would have been $2.5 million lower (December 31, 2024: other comprehensive loss $11.0 million higher).
We continued to incur losses in the current period. We recorded a net loss of $239.6 million for the year ended December 31, 2024, and net loss of $308.5 million in 2023, and a net loss of $126.8 million in 2022.
We continued to incur losses in the current period. We recorded a net loss of $199.0 million for the year ended December 31, 2025, and a net loss of $239.6 million and $308.5 million in the years ended December 31, 2024 and 2023, respectively.
See “Results of Operations” below for a discussion of the detailed components and analysis of the amounts above. Critical Accounting Policies and Estimates ​ In preparing our consolidated financial statements in accordance with U.S.
Refer to “ Results of Operations ” below for a discussion of the detailed components and analysis of the amounts above. Critical Accounting Policies and Estimates ​ In preparing our consolidated financial statements in accordance with U.S.
An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability. 90 ​ Table of Contents ● We need to regularly update our estimate of when a milestone is expected to be achieved.
An increase in the discount rate reduces the fair value of the contingent consideration liability whereas a decrease in the discount rate increases the fair market value of the contingent consideration liability. 95 ​ Table of Contents ● We regularly update our estimates of when milestones are expected to be achieved.
The amounts payable in accordance with the share and purchase agreement (“SPA”) are contingent upon realization of certain milestones associated with AMT-260. Contingent consideration was measured at fair value at the Acquisition Date with changes in fair value recognized in the consolidated statements of operations in research and development expenses.
The amounts payable in accordance with the related share and purchase agreement are contingent upon the achievement of certain milestones associated with AMT-260. Contingent consideration was measured at fair value as of the date of the uniQure France Acquisition, with changes in fair value subsequently recognized in the Consolidated Statements of Operations and Comprehensive Loss, within Research and development expenses.
Costs incurred in the year ended December 31, 2022 primarily related to our preclinical activities. ​ Preclinical programs & platform development ​ In the years ended December 31, 2024, 2023 and 2022 we incurred $3.9 million, $11.0 million and $7.2 million expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects. ​ Hemophilia B (AMT-061) We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.
During the year ended December 31, 2023, we incurred expenses of $10.0 million related to the upfront consideration paid to Apic Bio for the global licensing rights to AMT-162. ​ Preclinical programs & platform development ​ In the years ended December 31, 2025, 2024 and 2023, we incurred $3.6 million, $3.9 million and $11.0 million of expenses, respectively, related to our preclinical activities associated with product candidates for various other research programs and technology innovation projects. ​ Hemophilia B (AMT-061) We transitioned activities related to the clinical trial and long-term follow-up of patients to CSL Behring in December 2022.
CSL Behring’s minimum purchase commitments to us over the next 12 months are $13.6 million. ​ 101 ​ Table of Contents Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027.
CSL Behring’s minimum purchase commitments to us over the next 12 months are $16.4 million, of which $16.2 million relates to such minimum purchase commitments to Genezen. ​ Our DMSA with Genezen requires us to take or pay contract development services during a three-year period ending July 2027.
If as of December 31, 2024, we had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 33.7 million ($35.0 million).
If, as of December 31, 2025, the Company had assumed a 100% likelihood of AMT-260 advancing into a Phase III clinical study, then the fair value of the contingent consideration would have increased to EUR 51.4 million ($60.4 million).
Net cash generated from / (used in) investing activities ​ In 2024, we generated $163.0 million from our investing activities compared to using $205.7 million in 2023 and $182.7 million in 2022. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 2022 ​ ​ (in thousands) Proceeds from maturity of debt securities ​ $ 534,498 ​ $ 167,907 ​ ​ — Investment in debt securities ​ ​ (359,841) ​ ​ (366,439) ​ $ (163,146) Divestment of commercial manufacturing facility ​ ​ (8,321) ​ ​ — ​ ​ — Capital expenditures - European sites ​ ​ (1,783) ​ ​ (3,389) ​ ​ (11,904) Capital expenditures - Lexington site ​ ​ (1,585) ​ ​ (3,765) ​ ​ (5,784) Acquisition of uniQure France SAS, net of cash acquired ​ ​ — ​ ​ — ​ ​ (1,900) Net cash generated from / (used in) investing activities ​ $ 162,968 ​ $ (205,686) ​ $ (182,734) ​ ​ 104 ​ Table of Contents Net cash (used in) / generated from financing activities ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ 2024 2023 2022 ​ ​ (in thousands) Cash flows from financing activities ​ ​ ​ ​ ​ ​ ​ ​ ​ Proceeds from Royalty Financing Agreement, net of debt issuance costs ​ $ - ​ $ 370,062 ​ $ - Proceeds from issuance of shares related to employee stock option and purchase plans ​ ​ 2,123 ​ ​ 308 ​ ​ 1,445 Repayment of long-term debt ​ ​ (53,050) ​ ​ - ​ ​ - Contingent consideration milestone payment ​ ​ (8,559) ​ ​ (7,649) ​ ​ - Net cash (used in) / generated from financing activities ​ $ (59,486) ​ $ 362,721 ​ $ 1,445 ​ In December 2024, following the dosing of the first patient in Phase I/II clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France SAS based on contractually defined milestones.
Net cash (used in) / generated from investing activities ​ In 2025, we used $321.6 million in our investing activities, compared to $163.0 million generated from investing activities in 2024 and $205.7 million used in investing activities in 2023. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 ​ ​ (in thousands) Cash flows from investing activities ​ ​ ​ ​ ​ ​ ​ ​ ​ Proceeds from maturity of debt securities ​ $ 337,195 ​ $ 534,498 ​ $ 167,907 Investment in debt securities ​ ​ (658,375) ​ ​ (359,841) ​ ​ (366,439) Capital expenditures - European sites ​ ​ (406) ​ ​ (1,783) ​ ​ (3,389) Capital expenditures - Lexington sites ​ ​ (33) ​ ​ (1,585) ​ ​ (3,765) Divestment of commercial manufacturing facility ​ ​ — ​ ​ (8,321) ​ ​ — Net cash (used in) / generated from investing activities ​ $ (321,619) ​ $ 162,968 ​ $ (205,686) ​ Net cash generated from / (used in) financing activities ​ In 2025, we generated $415.4 million from our financing activities compared to $59.5 million used in financing activities in 2024 and $362.7 million generated from financing activities in 2023. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 ​ ​ (in thousands) Cash flows from financing activities ​ ​ ​ ​ ​ ​ ​ ​ ​ Proceeds from follow-on public offerings of ordinary shares, net of issuance costs ​ $ 380,737 ​ $ — ​ $ — Proceeds from issuance of pre-funded warrants, net of issuance costs ​ ​ 23,499 ​ ​ — ​ ​ — Proceeds from issuance of ordinary shares related to employee stock options and purchase plans ​ ​ 11,162 ​ ​ 2,123 ​ ​ 308 Repayment of long-term debt ​ ​ — ​ ​ (53,050) ​ ​ — Contingent consideration milestone payments ​ ​ — ​ ​ (8,559) ​ ​ (7,649) Proceeds from royalty financing agreement, net of issuance costs ​ ​ — ​ ​ — ​ ​ 370,062 Net cash generated from / (used in) financing activities ​ $ 415,398 ​ $ (59,486) ​ $ 362,721 ​ In December 2024, following the dosing of the first patient in Phase I/IIa clinical trial for AMT-260, we made a payment of EUR 30.0 million ($31.5 million) to the former shareholders of uniQure France based on contractually defined milestones.
The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024.
The increase of $5.0 million in external cost in 2024 compared to 2023 is a result of enrolling patients into a third cohort between November 2023 and December 2024 as well as expenses we incurred associated with the RMAT application and in preparation of the Type B meeting with the FDA in November 2024. ​ Temporal lobe epilepsy (AMT-260) In the years ended December 31, 2025, 2024 and 2023, we incurred $9.8 million, $12.1 million and $13.0 million of expenses, respectively.
In addition, our pledge of assets as collateral to secure our obligations under the 2024 Amended Facility may limit our ability to obtain debt financing.
In addition, our pledge of assets as collateral to secure our obligations under the senior secured term loan facility with Hercules may limit our ability to obtain debt financing.
We determined that we are an agent in the sale of HEMGENIX® to CSL Behring.
We arrange for HEMGENIX to be provided by Genezen to CSL Behring. We determined that we are an agent in the sale of HEMGENIX to CSL Behring.
We transferred these activities to Genezen as part of the Lexington Transaction. Following the Closing of the Lexington Transaction, title to HEMGENIX® supply directly passes from the contract manufacturer, Genezen, to CSL Behring. We do not control HEMGENIX® before it is transferred to CSL Behring. We arrange for HEMGENIX® to be provided by Genezen to CSL Behring.
We transferred these activities to Genezen as part of the Lexington Transaction, which closed on July 22, 2024 (“the Closing”). Following the Closing of the Lexington Transaction, title to HEMGENIX supply directly passes from the contract manufacturer, Genezen, to CSL Behring. We do not control HEMGENIX before it is transferred to CSL Behring.
We recognized interest income of $21.4 million in 2024, $19.6 million in 2023 and $0.6 million in 2022. Our interest income increased in 2024 by $1.9 million compared to 2023 primarily due to the interest income earned on investment securities and cash on hand.
We recognized interest income of $17.0 million in 2025, $21.4 million in 2024 and $19.6 million in 2023. Our interest income decreased in 2025 by $4.4 million compared to 2024, and increased in 2024 by $1.9 million compared to 2023, primarily due to market interest rate developments and fluctuating balances of investment securities and cash on hand year over year.
These are not allocated as they are deployed across multiple projects under development. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ 2024 ​ 2023 ​ 2022 ​ 2024 vs 2023 ​ 2023 vs 2022 ​ ​ (in thousands) Huntington's disease (AMT-130) ​ $ 17,989 ​ $ 12,991 ​ $ 19,846 ​ $ 4,998 ​ $ (6,855) Temporal lobe epilepsy (AMT-260) ​ ​ 12,053 ​ ​ 13,037 ​ ​ 16,199 ​ ​ (984) ​ ​ (3,162) Amyotrophic lateral sclerosis (AMT-162) ​ ​ 7,306 ​ ​ 16,039 ​ ​ — ​ ​ (8,733) ​ ​ 16,039 Fabry disease (AMT-191) ​ ​ 4,814 ​ ​ 2,659 ​ ​ 2,862 ​ ​ 2,155 ​ ​ (203) Programs in preclinical development and platform related expenses ​ ​ 3,927 ​ ​ 11,005 ​ ​ 7,157 ​ ​ (7,078) ​ ​ 3,848 Hemophilia B (AMT-061) ​ ​ — ​ ​ (1,336) ​ ​ 2,474 ​ ​ 1,336 ​ ​ (3,810) Total direct research and development expenses ​ $ 46,089 ​ $ 54,395 ​ $ 48,538 ​ $ (8,306) ​ $ 5,857 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Employee and contractor-related expenses ​ ​ 49,818 ​ ​ 74,514 ​ ​ 64,935 ​ ​ (24,696) ​ ​ 9,579 Facility expenses ​ ​ 21,627 ​ ​ 29,490 ​ ​ 23,582 ​ ​ (7,863) ​ ​ 5,908 Share-based compensation expense ​ ​ 9,295 ​ ​ 16,881 ​ ​ 18,402 ​ ​ (7,586) ​ ​ (1,521) Disposables ​ ​ 5,084 ​ ​ 13,232 ​ ​ 17,830 ​ ​ (8,148) ​ ​ (4,598) Severance costs ​ ​ 3,967 ​ ​ 2,188 ​ ​ — ​ ​ 1,779 ​ ​ 2,188 Impairment charge ​ ​ — ​ ​ 1,438 ​ ​ — ​ ​ (1,438) ​ ​ 1,438 Fair value changes related to contingent consideration ​ ​ (1,817) ​ ​ 15,895 ​ ​ 7,081 ​ ​ (17,712) ​ ​ 8,814 Other expenses ​ ​ 9,719 ​ ​ 6,831 ​ ​ 17,223 ​ ​ 2,888 ​ ​ (10,392) Total other research and development expenses ​ $ 97,693 ​ $ 160,469 ​ $ 149,053 ​ $ (62,776) ​ $ 11,416 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total research and development expenses ​ $ 143,782 ​ $ 214,864 ​ $ 197,591 ​ $ (71,082) ​ $ 17,273 ​ Direct research and development expenses Huntington disease (AMT-130) ​ In the years ended December 31, 2024, 2023 and 2022, we incurred $18.0 million, $13.0 million and $19.8 million expenses respectively.
These are not allocated as they are deployed across multiple projects under development. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Years ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ ​ ​ 2023 ​ ​ ​ 2025 vs 2024 ​ ​ ​ 2024 vs 2023 ​ ​ (in thousands) Huntington's disease (AMT-130) ​ $ 42,462 ​ $ 17,989 ​ $ 12,991 ​ $ 24,473 ​ $ 4,998 Temporal lobe epilepsy (AMT-260) ​ ​ 9,751 ​ ​ 12,053 ​ ​ 13,037 ​ ​ (2,302) ​ ​ (984) Fabry disease (AMT-191) ​ ​ 6,827 ​ ​ 4,814 ​ ​ 2,659 ​ ​ 2,013 ​ ​ 2,155 Amyotrophic lateral sclerosis (AMT-162) ​ ​ 6,336 ​ ​ 7,306 ​ ​ 16,039 ​ ​ (970) ​ ​ (8,733) Programs in preclinical development and platform related expenses ​ ​ 3,611 ​ ​ 3,927 ​ ​ 11,005 ​ ​ (316) ​ ​ (7,078) Hemophilia B (AMT-061) ​ ​ — ​ ​ — ​ ​ (1,336) ​ ​ — ​ ​ 1,336 Total direct research and development expenses ​ $ 68,987 ​ $ 46,089 ​ $ 54,395 ​ $ 22,898 ​ $ (8,306) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Employee and contractor-related expenses ​ ​ 35,268 ​ ​ 49,818 ​ ​ 74,514 ​ ​ (14,550) ​ ​ (24,696) Facility expenses ​ ​ 15,173 ​ ​ 21,627 ​ ​ 29,490 ​ ​ (6,454) ​ ​ (7,863) Share-based compensation expense ​ ​ 7,579 ​ ​ 9,295 ​ ​ 16,881 ​ ​ (1,716) ​ ​ (7,586) Fair value changes related to contingent consideration ​ ​ 6,247 ​ ​ (1,817) ​ ​ 15,895 ​ ​ 8,064 ​ ​ (17,712) Disposables ​ ​ 2,585 ​ ​ 5,084 ​ ​ 13,232 ​ ​ (2,499) ​ ​ (8,148) Severance costs ​ ​ — ​ ​ 3,967 ​ ​ 2,188 ​ ​ (3,967) ​ ​ 1,779 Impairment charge ​ ​ — ​ ​ — ​ ​ 1,438 ​ ​ — ​ ​ (1,438) Other expenses ​ ​ 4,834 ​ ​ 9,719 ​ ​ 6,831 ​ ​ (4,885) ​ ​ 2,888 Total other research and development expenses ​ $ 71,686 ​ $ 97,693 ​ $ 160,469 ​ $ (26,007) ​ $ (62,776) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Total research and development expenses ​ $ 140,673 ​ $ 143,782 ​ $ 214,864 ​ $ (3,109) ​ $ (71,082) ​ Direct research and development expenses Huntington disease (AMT-130) ​ In the years ended December 31, 2025, 2024 and 2023, we incurred $42.5 million, $18.0 million and $13.0 million of expenses, respectively.
As of December 31, 2024, our remaining minimum purchase commitments to Genezen amount to $36.5 million, with $15.8 million to be paid within the next 12 months.
As of December 31, 2025, our remaining minimum purchase commitments to Genezen amount to $16.2 million, with $10.3 million to be paid within the next 12 months.
In 2024, we recognized a net foreign currency loss of $10.5 million related to our borrowings from Hercules, the May 2023 royalty financing agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group.
We recognize foreign exchange results related to changes in these foreign currencies. In 2025, we recognized a net foreign currency gain of $26.1 million related to our borrowings from Hercules, the Royalty Financing Agreement and our cash and cash equivalents and investment securities as well as loans between entities within the uniQure group.
We also have short-term investment securities in U.S. and European government bonds maturing within two to five months. Our investment policy requires us to invest in U.S. and European government bonds with the highest investment credit rating.
The deposits are neither impaired nor past due. ​ We also have short-term investment securities in U.S. and European government bonds maturing within one to nine months. Our investment policy requires us to invest in U.S. and European government bonds with a high investment credit rating.
Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen. This reduced our fixed lease payment obligations by $20.7 million. We continue to guarantee such payments until the end of the lease term in May 2029.
Following the Closing of the Lexington Transaction, we assigned our lease for the Lexington facility to Genezen, which reduced our fixed lease payment obligations by $20.7 million.
We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX® have been earned. We recognized $10.1 million, $2.8 million and $100.0 million of license revenue for the years ended December 31, 2024, 2023 and 2022, respectively.
We recognize license revenue in relation to the License Sale when it becomes probable that regulatory and sales milestone events will be achieved as well as when royalties on sales of HEMGENIX have been earned.
Selling, general and administrative expenses for the year ended December 31, 2024 were $52.7 million, compared to $74.6 million and $55.1 million for the years ended December 31, 2023 and 2022, respectively. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Year ended December 31, ​ ​ 2024 ​ 2023 ​ 2022 ​ 2024 vs 2023 ​ 2023 vs 2022 ​ ​ (in thousands) Employee and contractor-related expenses ​ $ 21,343 ​ $ 24,399 ​ $ 21,065 ​ $ (3,056) ​ $ 3,334 Share-based compensation expense ​ ​ 11,928 ​ ​ 17,386 ​ ​ 15,479 ​ ​ (5,458) ​ ​ 1,907 Professional fees ​ ​ 7,261 ​ ​ 11,673 ​ ​ 7,133 ​ ​ (4,412) ​ ​ 4,540 Depreciation and facility costs ​ ​ 2,110 ​ ​ 2,051 ​ ​ 1,181 ​ ​ 59 ​ ​ 870 Intellectual property fees ​ ​ 1,744 ​ ​ 3,819 ​ ​ 1,480 ​ ​ (2,075) ​ ​ 2,339 Severance costs ​ ​ 1,223 ​ ​ 361 ​ ​ — ​ ​ 862 ​ ​ 361 Information technology costs ​ ​ 1,101 ​ ​ 6,526 ​ ​ 3,190 ​ ​ (5,425) ​ ​ 3,336 Financial advisory fees ​ ​ — ​ ​ 3,800 ​ ​ 1,000 ​ ​ (3,800) ​ ​ 2,800 Other expenses ​ ​ 5,947 ​ ​ 4,576 ​ ​ 4,531 ​ ​ 1,371 ​ ​ 45 Total selling, general and administrative expenses ​ $ 52,657 ​ $ 74,591 ​ $ 55,059 ​ $ (21,934) ​ $ 19,532 ​ ● We incurred $21.3 million in personnel and contractor expenses in the year ended December 31, 2024 compared to $24.4 million in 2023 and $21.4 million in 2022.
Selling, general and administrative expenses for the year ended December 31, 2025 were $65.5 million, compared to $52.7 million and $74.6 million for the years ended December 31, 2024 and 2023, respectively. ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Years ended December 31, ​ ​ ​ ​ 2025 ​ ​ ​ 2024 ​ 2023 ​ ​ ​ 2025 vs 2024 ​ ​ ​ 2024 vs 2023 ​ ​ (in thousands) Employee and contractor-related expenses ​ $ 24,941 ​ $ 21,343 ​ $ 24,399 ​ $ 3,598 ​ $ (3,056) Professional fees ​ ​ 16,638 ​ ​ 7,261 ​ ​ 11,673 ​ ​ 9,377 ​ ​ (4,412) Share-based compensation expense ​ ​ 10,100 ​ ​ 11,928 ​ ​ 17,386 ​ ​ (1,828) ​ ​ (5,458) Intellectual property fees ​ ​ 1,944 ​ ​ 1,744 ​ ​ 3,819 ​ ​ 200 ​ ​ (2,075) Depreciation and facility costs ​ ​ 1,910 ​ ​ 2,110 ​ ​ 2,051 ​ ​ (200) ​ ​ 59 Information technology costs ​ ​ 1,129 ​ ​ 1,101 ​ ​ 6,526 ​ ​ 28 ​ ​ (5,425) Severance costs ​ ​ — ​ ​ 1,223 ​ ​ 361 ​ ​ (1,223) ​ ​ 862 Financial advisory fees ​ ​ — ​ ​ — ​ ​ 3,800 ​ ​ — ​ ​ (3,800) Other expenses ​ ​ 8,794 ​ ​ 5,947 ​ ​ 4,576 ​ ​ 2,847 ​ ​ 1,371 Total selling, general and administrative expenses ​ $ 65,456 ​ $ 52,657 ​ $ 74,591 ​ $ 12,799 ​ $ (21,934) 102 ​ Table of Contents ​ ● We incurred $24.9 million in employee and contractor-related expenses in the year ended December 31, 2025 compared to $21.3 million in 2024 and $24.4 million in 2023.
Res e arch and development expenses We expense research and development (“R&D”) expenses as incurred. R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development.
R&D expenses include costs which relate to our primary activities of biopharmaceutical research and development.
Interest expense decreased by $1.7 million in 2024, compared to 2023, primarily due to the $50.0 million repayment of Hercules debt in July 2024, as well as a decrease in market interest rates. Interest expense increased by $2.9 million in 2023 compared to 2022 due to an increase in market interest rates related to the Hercules debt.
Interest expenses decreased by $6.0 million in 2025 compared to 2024, primarily due to the $50.0 million repayment of the Hercules debt in July 2024, as well as a decrease in market interest rates.
The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees.
The decrease of $2.1 million in 2024, compared to 2023, is primarily due to a decrease in professional fees; ● We incurred nil severance costs in the year ended December 31, 2025.
The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the Lexington Transaction, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of long-term incentive awards granted, partially offset by an increase in long-term incentive awards granted and costs associated with performance share units, granted in December 2021, for which achievement of performance criteria was deemed probable in 2024.
The decrease in 2024 of $5.5 million, compared to 2023, was primarily related to the divestment of our commercial manufacturing activities in July 2024, the October 2023 and August 2024 reorganizations, and a decrease in the fair value of granted long-term incentive awards, partially offset by an increase in granted long-term incentive awards and costs associated with performance share units, which were granted in December 2021, and for which achievement of performance criteria was deemed probable in 2024; ● We incurred $1.9 million in intellectual property fees including registration and professional fees in the year ended December 31, 2025 compared to $1.7 million in 2024 and $3.8 million in 2023.

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